Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2

Fast robust automated brain extraction

Smith, S. M. (2002) · Human Brain Mapping · doi:10.1002/hbm.10062 · Cited by 10,496 · see citing works

Segmentation of brain MR images through a hidden Markov random field model and the expectation-maximization algorithm

Zhang, Y., Brady, M., & Smith, S. M. (2001) · IEEE Transactions on Medical Imaging · doi:10.1109/42.906424 · Cited by 7,628 · see citing works

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520 · see citing works

Lysosomal storage diseases

Platt, F. M., d’Azzo, A., Davidson, B. L., Neufeld, E. F., & Tifft, C. J. (2018) · Nature Reviews Disease Primers · doi:10.1038/s41572-018-0025-4 · Cited by 1,052 · see citing works

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.107 · Cited by 954 · see citing works

LYSOSOMAL STORAGE DISEASES

Neufeld, E. F. (1991) · Annual Review of Biochemistry · doi:10.1146/annurev.bi.60.070191.001353 · Cited by 924 · see citing works

Lysosomal Storage Diseases

Pastores, G. M. (2012) · Elsevier eBooks · doi:10.1016/b978-1-4377-0435-8.00036-6 · Cited by 643 · see citing works

Association of Mutations in a Lysosomal Protein with Classical Late-Infantile Neuronal Ceroid Lipofuscinosis

Sleat, D. E., DONNELLY, R. J., Lackland, H., Liu, C., Sohár, I., Pullarkat, R. K., & Lobel, P. (1997) · Science · doi:10.1126/science.277.5333.1802 · Cited by 581 · see citing works

Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Schulz, A., Ajayi, T., Specchio, N., de Los Reyes, E., Gissen, P., Ballon, D., Dyke, J. P., Cahan, H., Slasor, P., Jacoby, D., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1712649 · Cited by 489 · see citing works

The effects of intracranial volume adjustment approaches on multiple regional MRI volumes in healthy aging and Alzheimer's disease

Voevodskaya, O., Simmons, A., Nordenskjöld, R., Kullberg, J., Ahlström, H., Lind, L., Wahlund, L., Larsson, E., Westman, E., & Initiative, A. D. N. (2014) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2014.00264 · Cited by 473 · see citing works

Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

Cearley, C. N., & Wolfe, J. H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.015 · Cited by 409 · see citing works

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA

Worgall, S., Sondhi, D., Hackett, N. R., Kosofsky, B., Kekatpure, M. V., Neyzi, N., Dyke, J. P., Ballon, D., Heier, L., Greenwald, B. M., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.022 · Cited by 402 · see citing works

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Gray, S. J., Nagabhushan Kalburgi, S., McCown, T. J., & Jude Samulski, R. (2013) · Gene Therapy · doi:10.1038/gt.2012.101 · Cited by 377 · see citing works

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

Hudry, E., & Vandenberghe, L. H. (2019) · Neuron · doi:10.1016/j.neuron.2019.02.017 · Cited by 368 · see citing works

The Neuronal Ceroid-Lipofuscinoses

Haltia, M. (2003) · Journal of Neuropathology & Experimental Neurology · doi:10.1093/jnen/62.1.1 · Cited by 354 · see citing works

Neuronal ceroid lipofuscinoses

Jalanko, A., & Braulke, T. (2008) · Biochimica et Biophysica Acta (BBA) - Molecular Cell Research · doi:10.1016/j.bbamcr.2008.11.004 · Cited by 339 · see citing works

Update of the mutation spectrum and clinical correlations of over 360 mutations in eight genes that underlie the neuronal ceroid lipofuscinoses

Kousi, M., Lehesjoki, A., & Mole, S. (2011) · Human Mutation · doi:10.1002/humu.21624 · Cited by 330 · see citing works

Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306 · see citing works

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

Samaranch, L., Salegio, E. A., San Sebastian, W., Kells, A. P., Foust, K. D., Bringas, J. R., Lamarre, C., Forsayeth, J., Kaspar, B. K., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.200 · Cited by 299 · see citing works

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291 · see citing works

Erratum: Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248 · see citing works

Use of the Mullen Scales of Early Learning for the Assessment of Young Children with Autism Spectrum Disorders

Akshoomoff, N. A. (2006) · Child Neuropsychology · doi:10.1080/09297040500473714 · Cited by 238 · see citing works

Inborn Errors of Mucopolysaccharide Metabolism: Faulty degradative mechanisms are implicated in this group of human diseases.

Neufeld, E. F., & Fratantoni, J. C. (1970) · Science · doi:10.1126/science.169.3941.141 · Cited by 215 · see citing works

Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis

Mole, S., Anderson, G. W., Band, H., Berkovic, S. F., Cooper, J. D., Holthaus, S.-M. K., McKay, T. R., Medina, D. L., Rahim, A. A., Schulz, A., et al. (2018) · The Lancet Neurology · doi:10.1016/s1474-4422(18)30368-5 · Cited by 206 · see citing works

Reliability and validity of the Infant and Toddler Quality of Life Questionnaire (ITQOL) in a general population and respiratory disease sample

Raat, H., Landgraf, J. M., Oostenbrink, R., Moll, H. A., & Essink-Bot, M.-L. (2007) · Quality of Life Research · doi:10.1007/s11136-006-9134-8 · Cited by 187 · see citing works

Therapeutic landscape for Batten disease: current treatments and future prospects

Johnson, T. B., Cain, J. T., White, K. A., Ramirez‐Montealegre, D., Pearce, D. A., & Weimer, J. M. (2019) · Nature Reviews Neurology · doi:10.1038/s41582-019-0138-8 · Cited by 187 · see citing works

Late infantile neuronal ceroid lipofuscinosis: Quantitative description of the clinical course in patients with CLN2 mutations

Steinfeld, R., Heim, P., von Gregory, H., Meyer, K., Ullrich, K., Goebel, H. H., & Kohlschütter, A. (2002) · American Journal of Medical Genetics · doi:10.1002/ajmg.10660 · Cited by 179 · see citing works

Mutational Analysis of the Defective Protease in Classic Late-Infantile Neuronal Ceroid Lipofuscinosis, a Neurodegenerative Lysosomal Storage Disorder

Sleat, D. E., Gin, R. M., Sohár, I., Wisniewski, K. E., Sklower-Brooks, S., Pullarkat, R. K., Palmer, D. N., Lerner, T. J., Boustany, R.-M., Uldall, P. V., et al. (1999) · The American Journal of Human Genetics · doi:10.1086/302427 · Cited by 177 · see citing works

Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector

Sondhi, D., Hackett, N. R., Peterson, D. A., Stratton, J., Baad, M., Travis, K. M., Wilson, J. M., & Crystal, R. G. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300049 · Cited by 170 · see citing works

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

Rosenberg, J. B., Kaplitt, M. G., De, B. P., Chen, A., Flagiello, T., Salami, C., Pey, E., Zhao, L., Ricart Arbona, R. J., Monette, S., et al. (2018) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2017.231 · Cited by 169 · see citing works

Adeno-associated virus (AAV) gene therapy for neurological disease

Weinberg, M. S., Samulski, R. J., & McCown, T. J. (2012) · Neuropharmacology · doi:10.1016/j.neuropharm.2012.03.004 · Cited by 154 · see citing works

Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

Nickel, M., Simonati, A., Jacoby, D., Lezius, S., Kilian, D., Van de Graaf, B., Pagovich, O. E., Kosofsky, B., Yohay, K., Downs, M., et al. (2018) · The Lancet Child &amp; Adolescent Health · doi:10.1016/s2352-4642(18)30179-2 · Cited by 148 · see citing works

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

Katz, M. L., Tecedor, L., Chen, Y., Williamson, B. G., Lysenko, E., Wininger, F. A., Young, W. M., Johnson, G. C., Whiting, R. E. H., Coates, J. R., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aac6191 · Cited by 134 · see citing works

Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis

Crystal, R. G., Sondhi, D., Hackett, N. R., Kaminsky, S. M., Worgall, S., Stieg, P. E., Souweidane, M. M., Hosain, S. A., Heier, L., Ballon, D., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.1131 · Cited by 129 · see citing works

High levels of persistent expression of α1-antitrypsin mediated by the nonhuman primate serotype rh. 10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

De, B. P., Heguy, A., Hackett, N. R., Ferris, B., Leopold, P. L., Lee, J., Pierre, L., Gao, G., Wilson, J. M., & Crystal, R. G. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.09.003 · Cited by 129 · see citing works

Neonatal Intramuscular Injection with Recombinant Adeno-Associated Virus Results in Prolonged beta-Glucuronidase Expression in Situ and Correction of Liver Pathology in Mucopolysaccharidosis Type VII Mice

Daly, T. M., Okuyama, T., Vogler, C. A., Haskins, M. E., Muzyczka, N., & Sands, M. S. (1999) · Human Gene Therapy · doi:10.1089/10430349950019219 · Cited by 127 · see citing works

Management Strategies for CLN2 Disease

Williams, R. E., Adams, H., Blohm, M. E. G., Cohen-Pfeffer, J. L., Reyes, E. D. L., Denecke, J., Drago, K., Fairhurst, C., Frazier, M., Guelbert, N. B., et al. (2017) · Pediatric Neurology · doi:10.1016/j.pediatrneurol.2017.01.034 · Cited by 124 · see citing works

Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses

Kohlschütter, A., Schulz, A., Bartsch, U., & Storch, S. (2019) · CNS Drugs · doi:10.1007/s40263-019-00620-8 · Cited by 124 · see citing works

Cerliponase Alfa: First Global Approval

Markham, A. (2017) · Drugs · doi:10.1007/s40265-017-0771-8 · Cited by 120 · see citing works

Neurological deterioration in late infantile neuronal ceroid lipofuscinosis

Worgall, S., Kekatpure, M. V., Heier, L., Ballon, D., Dyke, J. P., Shungu, D. C., Mao, X., Kosofsky, B. E., Kaplitt, M. G., Souweidane, M. M., et al. (2007) · Neurology · doi:10.1212/01.wnl.0000267885.47092.40 · Cited by 112 · see citing works

Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis

Fietz, M., Alsayed, M. D., Burke, D. G., Cohen-Pfeffer, J. L., Cooper, J. D., Dvořáková, L., Giugliani, R., Izzo, E., Jahnová, H., Lukács, Z., et al. (2016) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2016.07.011 · Cited by 107 · see citing works

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

Sondhi, D., Johnson, L. K., Purpura, K. P., Monette, S., Souweidane, M. M., Kaplitt, M. G., Kosofsky, B. E., Yohay, K. H., Ballon, D., Dyke, J. P., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.120 · Cited by 91 · see citing works

Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease

Cabrera-Salazar, M. A., Roskelley, E. M., Bu, J., Hodges, B. L., Yew, N. S., Dodge, J. C., Shihabuddin, L. S., Sohár, I., Sleat, D. E., Scheule, R. K., et al. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300249 · Cited by 82 · see citing works

Best practices for the use of intracerebroventricular drug delivery devices

Slavc, I., Cohen-Pfeffer, J. L., Gururangan, S., Krauser, J. M., Lim, D. A., Maldaun, M. V. C., Schwering, C., Shaywitz, A. J., & Westphal, M. (2018) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2018.05.003 · Cited by 78 · see citing works

Production and characterization of recombinant human CLN2 protein for enzyme-replacement therapy in late infantile neuronal ceroid lipofuscinosis

Lin, L., & Lobel, P. (2001) · Biochemical Journal · doi:10.1042/0264-6021:3570049 · Cited by 77 · see citing works

Mutation update: Review of TPP1 gene variants associated with neuronal ceroid lipofuscinosis CLN2 disease

Gardner, E., Bailey, M., Schulz, A., Aristorena, M., Miller, N., & Mole, S. (2019) · Human Mutation · doi:10.1002/humu.23860 · Cited by 72 · see citing works

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

Sondhi, D., Peterson, D. A., Edelstein, A., del Fierro, K., Hackett, N. R., & Crystal, R. G. (2008) · Experimental Neurology · doi:10.1016/j.expneurol.2008.04.022 · Cited by 68 · see citing works

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations

Souweidane, M. M., Fraser, J. F., Arkin, L. M., Sondhi, D., Hackett, N. R., Kaminsky, S. M., Heier, L., Kosofsky, B. E., Worgall, S., Crystal, R. G., et al. (2010) · Journal of Neurosurgery Pediatrics · doi:10.3171/2010.4.peds09507 · Cited by 66 · see citing works

Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS

Naidoo, J., Stanek, L. M., Ohno, K., Trewman, S., Samaranch, L., Hadaczek, P., O’Riordan, C. R., Sullivan, J., Sebastián, W. S., Bringas, J. R., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.07.008 · Cited by 59 · see citing works

Gene therapy and neurodevelopmental disorders

Gray, S. J. (2012) · Neuropharmacology · doi:10.1016/j.neuropharm.2012.06.024 · Cited by 58 · see citing works

Spectrum of Ocular Manifestations in CLN2-Associated Batten (Jansky-Bielschowsky) Disease Correlate with Advancing Age and Deteriorating Neurological Function

Orlin, A., Sondhi, D., Witmer, M. T., Wessel, M. M., Mezey, J. G., Kaminsky, S. M., Hackett, N. R., Yohay, K. H., Kosofsky, B. E., Souweidane, M. M., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0073128 · Cited by 54 · see citing works

Production and characterization of recombinant human CLN2 protein for enzyme-replacement therapy in late infantile neuronal ceroid lipofuscinosis

Li, L., & Lobel, P. (2001) · Biochemical Journal · doi:10.1042/bj3570049 · Cited by 50 · see citing works

An Adapted Clinical Measurement Tool for the Key Symptoms of CLN2 Disease

Wyrwich, K. W., Schulz, A., Nickel, M., Slasor, P., Ajayi, T., Jacoby, D. R., & Kohlschütter, A. (2018) · Journal of Inborn Errors of Metabolism and Screening · doi:10.1177/2326409818788382 · Cited by 45 · see citing works

Advances in the treatment of neuronal ceroid lipofuscinosis

Rosenberg, J. B., Chen, A., Kaminsky, S. M., Crystal, R. G., & Sondhi, D. (2019) · Expert Opinion on Orphan Drugs · doi:10.1080/21678707.2019.1684258 · Cited by 41 · see citing works

Complications related to the use of an intraventricular access device for the treatment of leptomeningeal metastases from solid tumor: a single centre experience in 112 patients

Zaïri, F., Rhun, É. L., Bertrand, N., Boulanger, T., Taillibert, S., Aboukaïs, R., Assaker, R., & Chamberlain, M. C. (2015) · Journal of Neuro-Oncology · doi:10.1007/s11060-015-1842-x · Cited by 40 · see citing works

Feasibility of Gene Therapy for Late Neuronal Ceroid Lipofuscinosis

Sondhi, D. (2001) · Archives of Neurology · doi:10.1001/archneur.58.11.1793 · Cited by 37 · see citing works

Reliability and Validity of the Child Health QuestionnairePF-50 for European Children with Cerebral Palsy

McCullough, N., Parkes, J. H., White‐Koning, M., Beckung, E., & Colver, A. F. (2007) · Journal of Pediatric Psychology · doi:10.1093/jpepsy/jsn048 · Cited by 28 · see citing works

Brain Region–Specific Degeneration with Disease Progression in Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2 Disease)

Dyke, J. P., Sondhi, D., Voss, H. U., Yohay, K. H., Hollmann, C., Mancenido, D., Kaminsky, S. M., Heier, L., Rudser, K. D., Kosofsky, B. E., et al. (2016) · American Journal of Neuroradiology · doi:10.3174/ajnr.a4669 · Cited by 26 · see citing works

Symmetric Age Association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease

Kovacs, K. D., Patel, S. C., Orlin, A., Kim, K., Everen, S. A. V., Conner, T., Sondhi, D., Kaminsky, S. M., D’Amico, D. J., Crystal, R. G., et al. (2020) · Ophthalmology Retina · doi:10.1016/j.oret.2020.01.011 · Cited by 25 · see citing works

Transduction Profile of the Marmoset Central Nervous System Using Adeno-Associated Virus Serotype 9 Vectors

Matsuzaki, Y., Konno, A., Mukai, R., Honda, F., Hirato, M., Yoshimoto, Y., & Hirai, H. (2016) · Molecular Neurobiology · doi:10.1007/s12035-016-9777-6 · Cited by 24 · see citing works

Assessment of Disease Severity in Late Infantile Neuronal Ceroid Lipofuscinosis Using Multiparametric MR Imaging

Dyke, J. P., Sondhi, D., Voss, H. U., Shungu, D. C., Mao, X., Yohay, K. H., Worgall, S., Hackett, N. R., Hollmann, C., Yeotsas, M., et al. (2012) · American Journal of Neuroradiology · doi:10.3174/ajnr.a3297 · Cited by 23 · see citing works

Expression and analysis of CLN2 variants in CHO cells: Q100r represents a polymorphism, and G389E and R447H represent loss-of-function mutations

Lin, L., & Lobel, P. (2001) · Human Mutation · doi:10.1002/humu.1170 · Cited by 14 · see citing works

Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses

Donsante, A., & Boulis, N. M. (2018) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2018.1492544 · Cited by 14 · see citing works

Neuronal ceroid-lipofuscinoses

Rakheja, D., & Bennett, M. J. (2018) · Translational Science of Rare Diseases · doi:10.3233/trd-180024 · Cited by 14 · see citing works

Advances in the treatment of neuronal ceroid lipofuscinosis

Sondhi, D., Rosenberg, J. B., de Graaf, B. G. V., Kaminsky, S. M., & Crystal, R. G. (2013) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2013.852081 · Cited by 9 · see citing works

Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3 year update from an ongoing multicenter extension study

Schulz, A., Specchio, N., Gissen, P., Reyes, E. D. L., Cahan, H., Slasor, P., Jacoby, D. R., & Ajayi, T. (2019) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2018.12.342 · Cited by 7 · see citing works

Persistent Treatment Effect of Cerliponase Alfa in Children with CLN2 Disease: A 3 Year Update from an Ongoing Multicenter Extension Study

Schulz, A., Specchio, N., Gissen, P., Reyes, E. D. L., Cahan, H., Slasor, P., Jacoby, D. R., & Ajayi, T. (2019) · Neuropediatrics · doi:10.1055/s-0039-1698182 · Cited by 7 · see citing works

Neuronal Ceroid-Lipofuscinoses

Rakheja, D., & Bennett, M. J. (2017) · IOS Press eBooks · doi:10.3233/978-1-61499-718-4-499 · Cited by 5 · see citing works

Investigative and emerging treatments for Batten disease

Tarczyluk, M. A., & Cooper, J. D. (2015) · Expert Opinion on Orphan Drugs · doi:10.1517/21678707.2015.1073148 · Cited by 4 · see citing works

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. F., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 895 · see citing works

Gene therapy advances: A meta-analysis of AAV usage in clinical settings

Au, H. K. E., Isalan, M., & Mielcarek, M. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2021.809118 · Cited by 284 · see citing works

AAV-based in vivo gene therapy for neurological disorders

Ling, Q., Herstine, J. A., Bradbury, A. M., & Gray, S. J. (2023) · Nature Reviews Drug Discovery · doi:10.1038/s41573-023-00766-7 · Cited by 206 · see citing works

rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis

Shen, W., Liu, S., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.1001263 · Cited by 173 · see citing works

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

Ferrari, S., Jacob, A., Cesana, D., Laugel, M., Beretta, S., Varesi, A., Unali, G., Conti, A., Canarutto, D., Albano, L., et al. (2022) · Cell stem cell · doi:10.1016/j.stem.2022.09.001 · Cited by 133 · see citing works

AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges

Kang, L., Jin, S., Wang, J., Lv, Z., Xin, C., Tan, C., Zhao, M., Wang, L., & Liu, J. (2023) · Journal of Controlled Release · doi:10.1016/j.jconrel.2023.01.067 · Cited by 131 · see citing works

Current clinical applications of AAV-mediated gene therapy

Byrne, B. J., Flanigan, K. M., Matesanz, S. E., Finkel, R. S., Waldrop, M. A., D'Ambrosio, E. S., Johnson, N. E., Smith, B. K., Bönnemann, C. G., Carrig, S., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.04.045 · Cited by 134 · see citing works

Neuronal Ceroid Lipofuscinosis: The Multifaceted Approach to the Clinical Issues, an Overview

Simonati, A., & Williams, R. E. (2022) · Frontiers in Neurology · doi:10.3389/fneur.2022.811686 · Cited by 88 · see citing works

Immune responses and immunosuppressive strategies for adeno-associated virus-based gene therapy for treatment of central nervous system disorders: current knowledge and approaches

Prasad, S., Dimmock, D. P., Greenberg, B., Walia, J. S., Sadhu, C., Tavakkoli, F., & Lipshutz, G. S. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.138 · Cited by 81 · see citing works

A versatile toolkit for overcoming AAV immunity

Li, X., Wei, X., Lin, J., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.991832 · Cited by 57 · see citing works

Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications

Ye, D., Chukwu, C., Yang, Y., Hu, Z., & Chen, H. (2024) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2024.115363 · Cited by 58 · see citing works

Drug delivery pathways to the central nervous system via the brain glymphatic system circumventing the blood‐brain barrier

Wang, X., Yin, Y., Zhou, H., Chi, B., Guan, L., Li, P., Li, J., & Wang, Y. (2024) · Exploration · doi:10.1002/exp.20240036 · Cited by 50 · see citing works

Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies

Stone, D., Aubert, M., & Jerome, K. R. (2023) · Gene Therapy · doi:10.1038/s41434-023-00405-1 · Cited by 41 · see citing works

Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement

Sevin, C., & Deiva, K. (2021) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2021.624988 · Cited by 38 · see citing works

Haploinsufficiency, Dominant Negative, and Gain-of-Function Mechanisms in Epilepsy: Matching Therapeutic Approach to the Pathophysiology

Carvill, G. L., Matheny, T., Hesselberth, J. R., & Demarest, S. (2021) · Neurotherapeutics · doi:10.1007/s13311-021-01137-z · Cited by 39 · see citing works

Delivery of CRISPR/Cas9 system by AAV as vectors for gene therapy

Wang, Y., Jiang, H., Li, M., Xu, Z., Xu, H., Chen, Y., Chen, K., Zheng, W., Lin, W., Lin, Z., et al. (2024) · Gene · doi:10.1016/j.gene.2024.148733 · Cited by 33 · see citing works

AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain

Costa-Verdera, H., Meneghini, V., Fitzpatrick, Z., Abou Alezz, M., Fabyanic, E., Huang, X., Dzhashiashvili, Y., Ahiya, A., Mangiameli, E., Valeri, E., et al. (2025) · Nature Communications · doi:10.1038/s41467-025-58778-3 · Cited by 32 · see citing works

Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?

Harkins, A. L., Ambegaokar, P. P., & Keeler, A. M. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00435 · Cited by 32 · see citing works

A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment

Horton, R. H., Saade, D. N., Markati, T., Harriss, E., Bönnemann, C. G., Francesco Muntoni, A., & Servais, L. (2022) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp-2022-329431 · Cited by 26 · see citing works

Neurosurgical gene therapy for central nervous system diseases

Patel, R. V., Nanda, P., & Richardson, R. M. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00434 · Cited by 23 · see citing works

Natural History Studies in NCL and Their Expanding Role in Drug Development: Experiences From CLN2 Disease and Relevance for Clinical Trials

Nickel, M., & Schulz, A. (2022) · Frontiers in Neurology · doi:10.3389/fneur.2022.785841 · Cited by 24 · see citing works

Precision medicine in epilepsy

McGinn, R. J., Stein, E. L. V., Stromberg, J. E. S., & Li, Y. (2022) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2022.04.001 · Cited by 24 · see citing works

Neuronal Ceroid Lipofuscinosis—Concepts, Classification, and Avenues for Therapy

Zhang, Y., Du, B., Zou, M., Peng, B., & Rao, Y. (2025) · CNS Neuroscience & Therapeutics · doi:10.1111/cns.70261 · Cited by 24 · see citing works

Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier

Critchley, B. J., Gaspar, H. B., & Benedetti, S. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.11.015 · Cited by 23 · see citing works

Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders

Limia, C. E. G., Baird, M., Schwartz, M., Saxena, S., Meyer, K., & Wein, N. (2022) · Journal of Personalized Medicine · doi:10.3390/jpm12121979 · Cited by 23 · see citing works

Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain

Stone, D., Aubert, M., & Jerome, K. R. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.09.001 · Cited by 22 · see citing works

Glial Dysfunction and Its Contribution to the Pathogenesis of the Neuronal Ceroid Lipofuscinoses

Takahashi, K., Nelvagal, H. R., Lange, J., & Cooper, J. D. (2022) · Frontiers in Neurology · doi:10.3389/fneur.2022.886567 · Cited by 21 · see citing works

An innate immune response to adeno-associated virus genomes decreases cortical dendritic complexity and disrupts synaptic transmission

Suriano, C. M., Kumar, N., Verpeut, J. L., Ma, J., Jung, C., Dunn, C. E., Carvajal, B. V., Nguyen, A. V., & Boulanger, L. M. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.03.036 · Cited by 21 · see citing works

Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders

Zhou, L., Wang, Y., Xu, Y., Zhang, Y., & Zhu, C. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101357 · Cited by 21 · see citing works

Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease)

Dulz, S., Schwering, C., Wildner, J., Spartalis, C., Schuettauf, F., Bartsch, U., Wibbeler, E., Nickel, M., Spitzer, M. S., Atiskova, Y., et al. (2022) · British Journal of Ophthalmology · doi:10.1136/bjo-2022-321260 · Cited by 20 · see citing works

Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model

Takahashi, K., Eultgen, E. M., Wang, S. H., Rensing, N. R., Nelvagal, H. R., Dearborn, J. T., Danos, O., Buss, N. A. P. S., Sands, M. S., Wong, M. L., et al. (2023) · Journal of Clinical Investigation · doi:10.1172/jci165908 · Cited by 20 · see citing works

Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Audouard, E., Oger, V., Meha, B., Cartier, N., Sevin, C., & Piguet, F. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2021.677895 · Cited by 18 · see citing works

An ERG and OCT study of neuronal ceroid lipofuscinosis CLN2 Battens retinopathy

Thompson, D., Handley, S. E., Henderson, R., Marmoy, O. R., & Gissen, P. (2021) · Eye · doi:10.1038/s41433-021-01594-y · Cited by 18 · see citing works

Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humans

Lüscher, C., Emiliani, V., Farahany, N. A., Gittis, A. H., Gradinaru, V., High, K. A., Roska, B., Sahel, J., Yizhar, O., Zeng, H., et al. (2025) · Nature Neuroscience · doi:10.1038/s41593-025-02097-9 · Cited by 21 · see citing works

Intravitreal gene therapy restores the autophagy-lysosomal pathway and attenuates retinal degeneration in cathepsin D-deficient mice

Liu, J., Bassal, M., Schlichting, S., Braren, I., Spiezio, A. D., Säftig, P., & Bartsch, U. (2022) · Neurobiology of Disease · doi:10.1016/j.nbd.2022.105628 · Cited by 16 · see citing works

CRISPR delivery systems for organ-specific targeting: Advances and challenges

Du, J., Wu, Q., Liu, C., Wang, N., & Gong, C. (2025) · Precision medicine and engineering. · doi:10.1016/j.preme.2025.100048 · Cited by 17 · see citing works

On the cusp of cures: Breakthroughs in Batten disease research

Brudvig, J. J., & Weimer, J. M. (2021) · Current Opinion in Neurobiology · doi:10.1016/j.conb.2021.08.003 · Cited by 14 · see citing works

An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice

Tecedor, L., Chen, Y. H., Leib, D. E., Ranum, P. T., Keiser, M. S., Lewandowski, B. C., Carrell, E. M., Лысенко, Е., Huerta-Ocampo, I., Arora, S., et al. (2025) · Science Translational Medicine · doi:10.1126/scitranslmed.adr2531 · Cited by 13 · see citing works

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

De, B. P., Rosenberg, J. B., Selvan, N., Wilson, I., Yusufzai, N., Greco, A., Kaminsky, S. M., Heier, L., Arbona, R. J. R., Miranda, I. C., et al. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.067 · Cited by 12 · see citing works

Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders

Wang, J., Gao, G., & Wang, D. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00391 · Cited by 10 · see citing works

Brain transcriptome analysis of a CLN2 mouse model as a function of disease progression

Domowicz, M. S., Chan, W., Vázquez, P. N. C., Gonzalez, T., & Schwartz, N. B. (2021) · Journal of Neuroinflammation · doi:10.1186/s12974-021-02302-z · Cited by 12 · see citing works

Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells

De, B. P., Cram, S., Lee, H., Rosenberg, J. B., Sondhi, D., Crystal, R. G., & Kaminsky, S. M. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.032 · Cited by 9 · see citing works

Evolving therapies in neuronopathic LSDs: opportunities and challenges

Rajan, D. S., & Escolar, M. L. (2022) · Metabolic Brain Disease · doi:10.1007/s11011-022-00939-0 · Cited by 10 · see citing works

Advancing the Research and Development of Enzyme Replacement Therapies for Lysosomal Storage Diseases

Puhl, A. C., & Ekins, S. (2022) · GEN Biotechnology · doi:10.1089/genbio.2021.0013 · Cited by 9 · see citing works

Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors

Rostami, M. R., Leopold, P. L., Vasquez, J. M., Rougvie, M. D. M., Al-Shakaki, A., Hssain, A. A., Robay, A., Hackett, N. R., Mezey, J. G., & Crystal, R. G. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.101136 · Cited by 5 · see citing works

Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases

Daci, R., Gray‐Edwards, H. L., Shazeeb, M. S., Vardar, Z., Vachha, B. A., Cataltepe, O. I., & Flotte, T. R. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.057 · Cited by 7 · see citing works

Automated Retinal Layer Segmentation in CLN2-Associated Disease: Commercially Available Software Characterizing a Progressive Maculopathy

Kovacs, K. D., Orlin, A., Sondhi, D., Kaminsky, S. M., D’Amico, D. J., Crystal, R. G., & Kiss, S. (2021) · Translational Vision Science & Technology · doi:10.1167/tvst.10.8.23 · Cited by 6 · see citing works

CLN7 gene therapy: hope for an ultra-rare condition

Brudvig, J. J., & Weimer, J. M. (2022) · Journal of Clinical Investigation · doi:10.1172/jci157820 · Cited by 6 · see citing works

The clinical toxicity of advanced therapy medicinal products

Henderson, F. J. H., & Hill, S. L. (2025) · British Journal of Clinical Pharmacology · doi:10.1002/bcp.70104 · Cited by 5 · see citing works

Phase I clinical trial of intracerebral injection of lentiviral-ABCD1 for the treatment of cerebral adrenoleukodystrophy

Wang, Q., Wang, J., Ling, Z., Cui, Z., Gong, J., Zhang, R., Li, S., Wang, Y., Yang, R., Huang, D., et al. (2024) · 中国科学通报：英文版 · doi:10.1016/j.scib.2024.04.072 · Cited by 5 · see citing works

Viral vectors for gene delivery to the central nervous system

Ingusci, S., Hall, B. L., Goins, W. F., Cohen, J. B., & Glorioso, J. C. (2024) · Handbook of clinical neurology · doi:10.1016/b978-0-323-90120-8.00001-0 · Cited by 5 · see citing works

Direct Convective Delivery for Nervous System Gene Therapy

Elder, J. B., & Lonser, R. R. (2024) · Neurosurgery Clinics of North America · doi:10.1016/j.nec.2024.09.002 · Cited by 5 · see citing works

Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies

Fogel, B. L., Klopstock, T., Lynch, D. R., Maltecca, F., Verma, M., Minassian, B. A., Platt, F. M., Gonçalves, D. F., Puccio, H. M., Roos, A., et al. (2025) · Annals of Neurology · doi:10.1002/ana.27271 · Cited by 5 · see citing works

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease

Sondhi, D., Kaminsky, S. M., Rosenberg, J. B., Rostami, M. R., Hackett, N. R., & Crystal, R. G. (2025) · Human Gene Therapy · doi:10.1089/hum.2024.182 · Cited by 5 · see citing works

Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey

Corazolla, E. M., Eskes, E. C. B., Veldwijk, J., Brands, M. M. M. G., Dekker, H., van de Mheen, E., Langeveld, M., Hollak, C. E. M., & Sjouke, B. (2024) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-024-03371-y · Cited by 4 · see citing works

Gene editing for Spinocerebellar ataxia type 3 taking advantage of the human ATXN3L paralog as replacement gene

Rybarikova, M., Rey, M. J., Hasanovic, E., Sipion, M., Rambousek, L., & Déglon, N. (2025) · Gene Therapy · doi:10.1038/s41434-025-00557-2 · Cited by 3 · see citing works

Genetic therapies for neurological diseases

Rahim, A. A., Kurian, M. A., Zhou, H., Ferguson, R., Tabrizi, S. J., Lignani, G., Aquilina, K., & Waddington, S. N. (2025) · Pharmacological Reviews · doi:10.1016/j.pharmr.2025.100093 · Cited by 4 · see citing works

AAV vectors trigger DNA damage responses and STING-dependent inflammation in human CNS cells

Costa-Verdera, H., Meneghini, V., Fitzpatrick, Z., Alezz, M. A., Fabyanic, E., Huang, X., Dzhashiashvili, Y., Ahiya, A., Mangiameli, E., Valeri, E., et al. (2024) · doi:10.21203/rs.3.rs-4171795/v1 · Cited by 32 · see citing works

MR ‐Guidance of Gene Therapy for Brain Diseases: Moving From Palliative Treatment to Cures

Bermudez, D. H., Lilieholm, T., & Block, W. F. (2025) · Journal of Magnetic Resonance Imaging · doi:10.1002/jmri.29804 · Cited by 2 · see citing works

Optimization of systemic AAV9 gene therapy in Niemann–Pick disease, type C1 mice

Mylvara, A. V., Gibson, A. L., Gu, T., Davidson, C. D., Incao, A. A., Melnyk, K. D., Gembic, S., Pierre-Jacques, D., Cologna, S. M., Venditti, C. P., et al. (2026) · Life Science Alliance · doi:10.26508/lsa.202402874 · Cited by 2 · see citing works

Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways

Rybarikova, M., Almacellas‐Barbanoj, A., Schorge, S., & Déglon, N. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.177 · Cited by 2 · see citing works

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 micePreprint

Mylvara, A. V., Gibson, A. L., Gu, T., Davidson, C. D., Incao, A. A., Melnyk, K. D., Gembic, S. R., Pierre-Jacques, D., Cologna, S. M., Venditti, C. P., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.06.07.597901 · Cited by 2 · see citing works

Gene Therapy for Spinal Cord Injury

Verhaagen, J., Sydney-Smith, J. D., Middelhoff, S., Butter, L. C., de Winter, F., & Bradbury, E. J. (2025) · Springer eBooks · doi:10.1007/978-3-031-89307-0_10 · Cited by 1 · see citing works

Advances in Gene Therapy for CLN2 (Batten) Disease

De, B. P., Rosenberg, J. B., Leopold, P. L., Kaminsky, S. M., Sondhi, D., & Crystal, R. G. (2025) · Springer eBooks · doi:10.1007/978-3-031-89307-0_9 · Cited by 1 · see citing works

Viral and non-viral cellular therapies for neurodegeneration

Srivastav, J., & Sharma, S. (2026) · Frontiers in Medicine · doi:10.3389/fmed.2025.1718669 · Cited by 1 · see citing works

Sensory neuronopathy secondary to gene therapy with AT845 for Pompe disease

Pena-Guzman, L., Bolano-Diaz, C., Lleixà, C., Querol, L., Trainor, C., Suetterlin, K. J., Straub, V. W., & Díaz‐Manera, J. (2026) · Neuromuscular Disorders · doi:10.1016/j.nmd.2026.106441 · Cited by 1 · see citing works

Gene delivery for cerebral neurodegenerative disorders: Current advancements and limitations

Dayma, K., & Upadhyay, A. (2026) · Molecular Therapy · doi:10.1016/j.ymthe.2026.06.030 · Cited by 1 · see citing works

Missense Variation in TPP1 Gene causes Neuronal Ceroid Lipofuscinosis Type 2 in a Family from Jammu and Kashmir-IndiaPreprint

Angural, A., Ponnusamy, K., Langeh, D., Kumari, M., Spolia, A., Rai, E., Sharma, A., Pandita, K. K., & Sharma, S. (2021) · Preprints.org · doi:10.20944/preprints202107.0661.v1 · Cited by 1 · see citing works

Natural history variations for neuronal ceroid lipofuscinosis type 2: In support of newborn screening

Miskimen, A. C. C., Cohen, L. L., Yozawitz, E. G., & Grinspan, Z. M. (2023) · Epilepsia · doi:10.1111/epi.17544 · Cited by 1 · see citing works

Cortical interneuron loss and seizure generation as novel clinically relevant disease phenotypes in Cln2 R207X micePreprint

Takahashi, K., Eultgen, E. M., Wang, S. H., Rensing, N. R., Nelvagal, H. R., Dearborn, J. T., Sands, M. S., Wong, M. L., & Cooper, J. D. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.03.11.483984

Additional file 4 of Brain transcriptome analysis of a CLN2 mouse model as a function of disease progression

Domowicz, M. S., Chan, W.-C., Vázquez, P. N. C., Gonzalez, T., & Schwartz, N. B. (2021) · Figshare · doi:10.6084/m9.figshare.16960276

Consequences of human genetic variations in KIAA0319L, encoding adeno-associated virus receptor, on AAV-mediated gene transfer

Vasquez, J. M., Mackey, H. L., Rostami, M. R., Leopold, P. L., & Crystal, R. G. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201709

Caregiver Reports of Neurodevelopmental Functions in Pediatric Lysosomal Storage Disorders: A Scoping Review

Gillan, E., Leiro, B., Nagy, A. M., Eichler, F., & Townsend, E. L. (2026) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.70179

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

De, B. P., Rosenberg, J. B., Selvan, N., Wilson, I., Yusufzai, N., Greco, A., Kaminsky, S. M., Heier, L., Arbona, R. J. R., Miranda, I. C., et al. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.067 · Cited by 12 · see citing works

Gene therapy for CLN2 disease

Norton, M. (2020) · Science · doi:10.1126/science.370.6521.1177-r

Long Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Non-human Primates for the Treatment of Late Infantile Neuronal Lipofuscinosis

Sondhi, D., Johnson, L. K., De, B. P., Janda, K. D., Souweidane, M. M., Kaplitt, M. G., Rosenberg, J., Moreno, A. Y., Pagovich, O. E., Koob, G. F., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hum.2012.120 · Cited by 1 · see citing works

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

Sondhi, D., Johnson, L. K., Purpura, K. P., Monette, S., Souweidane, M. M., Kaplitt, M. G., Kosofsky, B. E., Yohay, K. H., Ballon, D., Dyke, J. P., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.120 · Cited by 91 · see citing works

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA

Worgall, S., Sondhi, D., Hackett, N. R., Kosofsky, B., Kekatpure, M. V., Neyzi, N., Dyke, J. P., Ballon, D., Heier, L., Greenwald, B. M., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.022 · Cited by 402 · see citing works