Sondhi, Dolan

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

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 402

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

Virus-based gene therapy researchAlzheimer's disease research and treatments

Cited by 176

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

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 170

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 & Adolescent Health

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 148

Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models

Zhao, L., Gottesdiener, A. J., Parmar, M., Li, M., Kaminsky, S. M., Chiuchiolo, M. J., Sondhi, D., Sullivan, P. M., Holtzman, D. M., Crystal, R. G., et al. (2016) · Neurobiology of Aging

Alzheimer's disease research and treatmentsVirus-based gene therapy research

Cited by 107

Gene therapy for metachromatic leukodystrophy

Rosenberg, J. B., Kaminsky, S. M., Aubourg, P., Crystal, R. G., & Sondhi, D. (2016) · Journal of Neuroscience Research

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 91

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

Piguet, F., Sondhi, D., Piraud, M., Fouquet, F., Hackett, N. R., Ahouansou, O., Vanier, M.-T., Bieche, I., Aubourg, P., Crystal, R. G., et al. (2012) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 91

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

Sondhi, D., Kaminsky, S. M., Hackett, N. R., Pagovich, O. E., Rosenberg, J. B., De, B. P., Chen, A., Van de Graaf, B., Mezey, J. G., Mammen, G. W., et al. (2020) · Science Translational Medicine

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 72

Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates

Rosenberg, J. B., Sondhi, D., Rubin, D. G., Monette, S., Chen, A., Cram, S., De, B. P., Kaminsky, S. M., Sevin, C., Aubourg, P., et al. (2014) · Human Gene Therapy Clinical Development

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 58

Safety of Direct Administration of AAV2 CU hCLN2, a Candidate Treatment for the Central Nervous System Manifestations of Late Infantile Neuronal Ceroid Lipofuscinosis, to the Brain of Rats and Nonhuman Primates

Hackett, N. R., Redmond, D. E., Sondhi, D., Giannaris, E. L., Vassallo, E., Stratton, J., Qiu, J., Kaminsky, S. M., Lesser, M. L., Fisch, G. S., et al. (2005) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 57

Biology of the Adrenal Gland Cortex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders

Markmann, S., De, B. P., Reid, J., Jose, C. L., Rosenberg, J. B., Leopold, P. L., Kaminsky, S. M., Sondhi, D., Pagovich, O., & Crystal, R. G. (2018) · Human Gene Therapy

Virus-based gene therapy researchAdrenal Hormones and Disorders

Cited by 42

Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Rosenberg, J. B., Chen, A., De, B. P., Dyke, J. P., Ballon, D. J., Monette, S., Ricart Arbona, R. J., Kaminsky, S. M., Crystal, R. G., & Sondhi, D. (2021) · Human Gene Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 41

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

Sondhi, D., Stiles, K. M., De, B. P., & Crystal, R. G. (2017) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 36

AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in alzheimer's disease mice

Günaydin, C., Sondhi, D., Kaminsky, S. M., Lephart, H. C., Leopold, P. L., Hackett, N. R., Khanna, R., & Crystal, R. G. (2024) · Molecular Therapy

Alzheimer's disease research and treatmentsVirus-based gene therapy research

Cited by 26

365. Long-Term Toxicology Evaluation of AAVrh. 10hARSA Administration to the CNS of Nonhuman Primates to Treat Metachromatic Leukodystrophy

Sondhi, D., Rosenberg, J. B., De, B. P., Monette, S., Chen, A., Flagiello, T., Caffrey, E., Kaminsky, S. M., Aubourg, P., & Crystal, R. G. (2016) · Molecular Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 3