Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

Cited by (606)

Items 101–200 of 606

1. Stem cell therapeutics and gene therapy for neurologic disorders
   Kevin S. Chen, Emily J. Koubek, Stacey A. Sakowski, Eva L. Feldman · 2024 · Neurotherapeutics
2. Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque
   Diego A. Espinoza, Xing Fan, Di Yang, Stefan Cordes, Lauren L. Truitt, Katherine R. Calvo, Idalia M. Yabe, Selami Demirci, Kristin J. Hope, So Gun Hong et al. · 2019 · Molecular Therapy
3. Biomarker Identification, Safety, and Efficacy of High-Dose Antioxidants for Adrenomyeloneuropathy: a Phase II Pilot Study
   Carlos Casasnovas, Montserrat Ruíz, Agatha Schlüter, Alba Naudí, Stéphane Fourcade, Misericordia Veciana, Sara Castañer, Antonia Albertí, Núria Bargalló, Maria R. Johnson et al. · 2019 · Neurotherapeutics
4. Positron emission tomography reporter gene strategy for use in the central nervous system
   Tom Haywood, Corinne Beinat, Gayatri Gowrishankar, Chirag B. Patel, Israt S. Alam, Surya Murty, Sanjiv Sam Gambhir · 2019 · Proceedings of the National Academy of Sciences
5. The Landscape of Hematopoietic Stem Cell Transplant and Gene Therapy for X-Linked Adrenoleukodystrophy
   Eric James Mallack, Bela Rui Turk, Helena Yan, Florian Eichler · 2019 · Current Treatment Options in Neurology
6. Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing
   Jakob M. Domm, Sarah K. Wootton, Jeffrey A. Medin, Michael L. West · 2021 · Molecular Genetics and Metabolism
7. Genetic testing of leukodystrophies unraveling extensive heterogeneity in a large cohort and report of five common diseases and 38 novel variants
   Nejat Mahdieh, Mahdieh Soveizi, Ali Reza Tavasoli, Ali Rabbani, Mahmoud Reza Ashrafi, Alfried Kohlschütter, Bahareh Rabbani · 2021 · Scientific Reports
8. Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy
   Robert Chiesa, Jaap Jan Boelens, Christine Duncan, Jörn‐Sven Kühl, Caroline Sevin, Neena Kapoor, Vinod K. Prasad, Caroline A. Lindemans, Simon Allan Jones, Hernán M. Amartino et al. · 2021 · Blood Advances
9. Advances in Gene Therapy for Fanconi Anemia
   Paula Rı́o, Susana Navarro, Juan Antonio Bueren · 2018 · Human Gene Therapy
10. Metabolic rerouting via SCD1 induction impacts X-linked adrenoleukodystrophy
    Quentin Raas, Malu-Clair van de Beek, Sonja Forss‐Petter, Inge M. E. Dijkstra, Abigail Deschiffart, Briana C. Freshner, Tamara J. Stevenson, Yorrick R. J. Jaspers, Liselotte Nagtzaam, Ronald J. A. Wanders et al. · 2021 · Journal of Clinical Investigation
11. Enhancing pediatric access to cell and gene therapies
    Crystal Lee Mackall, Catherine M. Bollard, Nancy Goodman, Casey Carr, Rebecca Gardner, Rayne Helen Rouce, Elena Sotillo, Rich Stoner, Fyodor D. Urnov, Alan S. Wayne et al. · 2024 · Nature Medicine
12. A Scoping Review of Inborn Errors of Metabolism Causing Progressive Intellectual and Neurologic Deterioration (PIND)
    Hilde A. G. Warmerdam, Elise A. Ferreira, Laura A. Tseng, Jessica Y. Lee, Agnies M. van Eeghen, Carlos R. Ferreira, Clara van Karnebeek · 2020 · Frontiers in Neurology
13. Current landscape of vector safety and genotoxicity after hematopoietic stem or immune cell gene therapy
    Giorgio Ottaviano, Waseem Qasim · 2025 · Leukemia
14. Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1
    Caitlin J. VanLith, Rebekah M. Guthman, Clara T. Nicolas, Kari L. Allen, Zeji Du, Dong Jin Joo, Scott L. Nyberg, Joseph B. Lillegard, Raymond Hickey · 2018 · Human Gene Therapy
15. Gene therapy for blood diseases
    Kohn, Donald B · 2019 · Current Opinion in Biotechnology
16. Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement
    Caroline Sevin, Kumaran Deiva · 2021 · Frontiers in Molecular Biosciences
17. Ex vivo expansion of hematopoietic stem cells: Finally transitioning from the lab to the clinic
    Eran Zimran, Luena Papa, Ronald Hoffman · 2021 · Blood Reviews
18. Retroviral gene therapy in Germany with a view on previous experience and future perspectives
    Michael Alexander Morgan, Melanie Galla, Manuel Grez, Boris Fehse, Axel Schambach · 2021 · Gene Therapy
19. Genetic therapies for sickle cell disease
    Erica B. Esrick, Daniel E. Bauer · 2018 · Seminars in Hematology
20. The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome
    David R. Hampson, Alexander W.M. Hooper, Yosuke Niibori · 2019 · Brain Sciences
21. Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model.
    N. Uchida, Tina Nassehi, C. Drysdale, Jackson Gamer, Morgan Yapundich, Aylin C. Bonifacino, Allen E. Krouse, N. Linde, Matthew M. Hsieh, R. Donahue et al. · 2019 · Molecular Therapy
22. Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs
    Virginie Baylot, Thi Khanh Le, David Taïeb, Palma Rocchi, Laurence Colleaux · 2024 · Communications Biology
23. Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy
    Paul J. Orchard, David R. Nascene, Weston P. Miller, Ashish O. Gupta, Daniel L. Kenney‐Jung, Troy C. Lund · 2019 · Blood
24. CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation
    Alberto Daniel-Moreno, Andrés Lamsfus‐Calle, Janani Raju, Justin S. Antony, Rupert Handgretinger, Markus Mezger · 2019 · Bone Marrow Transplantation
25. Gene-Based Approaches to Inherited Neurometabolic Diseases
    Valentina Poletti, Alessandra Biffi · 2019 · Human Gene Therapy
26. Saturated very long-chain fatty acids regulate macrophage plasticity and invasiveness
    Bettina Zierfuss, Agnieszka Buda, Andrea Villoria-González, Maxime Logist, Jure Fabjan, Patricia Parzer, Claire Battin, Streggi Vandersteene, Inge M. E. Dijkstra, Petra Waidhofer‐Söllner et al. · 2022 · Journal of Neuroinflammation
27. Time‐restricted release of multiple neurotrophic factors promotes axonal regeneration and functional recovery after peripheral nerve injury
    Liangliang Huang, Bing Xia, Xiaowei Shi, Jianbo Gao, Yujie Yang, Feng Xu, Fengyu Qi, Chao Liang, Jinghui Huang, Zhuojing Luo · 2019 · The FASEB Journal
28. Strategies for Targeting Retroviral Integration for Safer Gene Therapy: Advances and Challenges
    Kristine E. Yoder, Anthony J. Rabe, Richard A. Fishel, Ross C. Larue · 2021 · Frontiers in Molecular Biosciences
29. Gene Therapy for Genetic Syndromes: Understanding the Current State to Guide Future Care
    Marian L. Henderson, Jacob K. Zieba, Xiaopeng Li, Daniel B. Campbell, Michael R. Williams, Daniel L. Vogt, Caleb P. Bupp, Yvonne M. Edgerly, Surender Rajasekaran, Nicholas Hartog et al. · 2024 · BioTech
30. Laboratory diagnosis of disorders of peroxisomal biogenesis and function: a technical standard of the American College of Medical Genetics and Genomics (ACMG)
    Irene De Biase, Silvia Tortorelli, Lisa E. Kratz, Steven J. Steinberg, Kristina P. Cusmano‐Ozog, Nancy Braverman · 2019 · Genetics in Medicine
31. Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing
    Kenneth G. Cornetta, Tsai‐Yu Lin, Danilo Pellin, Donald B. Kohn · 2022 · Molecular Therapy — Methods & Clinical Development
32. Newborn Screening for X-Linked Adrenoleukodystrophy: Review of Data and Outcomes in Pennsylvania
    Priestley, Jessica R. C., Adang, Laura A., Drewes Williams, Sarah, Lichter-Konecki, Uta, Menello, Caitlin, Engelhardt, Nicole M., DiPerna, James C., DiBoscio, Brenda, Ahrens-Nicklas, Rebecca C., Edmondson, Andrew C. et al. · 2022 · International Journal of Neonatal Screening
33. Therapeutic genetic restoration through allogeneic brain microglia replacement
    Marius Marc-Daniel Mader, Alexa Scavetti, Yongjin Yoo, Aaron Tianyue Chai, Takeshi Uenaka, Marius Wernig · 2025 · Nature
34. In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
    Sara Marcó, Virginia Haurigot, Fátima Bosch · 2019 · Human Gene Therapy
35. Childhood-onset hereditary spastic paraplegia and its treatable mimics
    Darius Ebrahimi‐Fakhari, Afshin Saffari, Phillip Lawrence Pearl · 2021 · Molecular Genetics and Metabolism
36. ABCD1 and X‐linked adrenoleukodystrophy: A disease with a markedly variable phenotype showing conserved neurobiology in animal models
    Joshua Manor, Hyung-Lok Chung, Pranjali Bhagwat, Michael F. Wangler · 2021 · Journal of Neuroscience Research
37. Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside
    Stéphanie Cherqui · 2021 · Cells
38. Nervonic Acid Attenuates Accumulation of Very Long-Chain Fatty Acids and is a Potential Therapy for Adrenoleukodystrophy
    Márcia R. Terluk, Julianne Tieu, Siddhee A. Sahasrabudhe, Ann B. Moser, Paul A. Watkins, Gerald V. Raymond, Reena V. Kartha · 2022 · Neurotherapeutics
39. Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy
    Isabelle Weinhofer, Paulus Stefan Rommer, Andreas Gleiß, Markus Ponleitner, Bettina Zierfuss, Petra Waidhofer‐Söllner, Stéphane Fourcade, Katharina Grabmeier‐Pfistershammer, Marie‐Christine Reinert, Jens Christian Göpfert et al. · 2023 · EBioMedicine
40. Successful intracranial delivery of trastuzumab by gene-therapy for treatment of HER2-positive breast cancer brain metastases
    Inbal Zafir‐Lavie, Shay Sherbo, Haim Goltsman, Felix Badinter, Eilam Yeini, Paula Ofek, Reem Miari, Osnat Tal, Atar Liran, Tamar Shatil et al. · 2018 · Journal of Controlled Release
41. Gene therapy for lysosomal storage diseases and peroxisomal diseases
    Ohashi, Toya · 2019 · Journal of Human Genetics
42. FDA advisers back gene therapy for rare form of blindness
    Heidi K. Ledford · 2017 · Nature
43. Targeting foam cell formation in inflammatory brain diseases by the histone modifier MS‐275
    Bettina Zierfuss, Isabelle Weinhofer, Agnieszka Buda, Niko P. Popitsch, Lena Hess, Verena Moos, Simon Hametner, Stephan Kemp, Wolfgang Köhler, Sonja Forss‐Petter et al. · 2020 · Annals of Clinical and Translational Neurology
44. Non-toxic HSC Transplantation-Based Macrophage/Microglia-Mediated GDNF Delivery for Parkinson’s Disease
    Cang Chen, Michael J. Guderyon, Yang Li, Guo Ge, Anindita Bhattacharjee, Cori Ballard, Zhixu He, Eliezer Masliah, Robert A. Clark, Jason C. O’Connor et al. · 2019 · Molecular Therapy — Methods & Clinical Development
45. In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery
    Cort B. Breuer, Killian S. Hanlon, Jeya-shree Natasan, Adrienn Volak, Amine Meliani, Federico Mingozzi, Benjamin P. Kleinstiver, James J. Moon, Casey A. Maguire · 2020 · Scientific Reports
46. Gene therapy for inherited metabolic diseases
    Berna Şeker Yılmaz, Sonam Gurung, Dany P.B. Perocheau, John Robert Counsell, Julien Baruteau · 2020 · PubMed Central
47. Reference standards for accurate validation and optimization of assays that determine integrated lentiviral vector copy number in transduced cells
    Barbara S. Paugh, Lajos Baranyi, Andre J Roy, Hua‐Jun He, Lindsay N. Harris, Kenneth D. Cole, Moria Artlip, Caroline Raimund, Patricia S. Langan, Srikanta Jana et al. · 2021 · Scientific Reports
48. A Microfluidic Device to Enhance Viral Transduction Efficiency During Manufacture of Engineered Cellular Therapies
    Nathan Moore, John R. Chevillet, Laura J. Healey, Connor P. McBrine, Daniel T. Doty, José A. L. Santos, Bryan Teece, James G. Truslow, Vienna L. Mott, Peter T. Hsi et al. · 2019 · Scientific Reports
49. Emerging cellular themes in leukodystrophies
    Joseph C. Nowacki, Ashley M. Fields, Meng‐meng Fu · 2022 · Frontiers in Cell and Developmental Biology
50. History of Adrenal Research: From Ancient Anatomy to Contemporary Molecular Biology
    Walter L. Miller, Perrin C. White · 2022 · Endocrine Reviews
51. Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia
    Anusha Sivakumar, Stéphanie Cherqui · 2022 · Frontiers in Genome Editing
52. Gene therapy in pediatrics – Clinical studies and approved drugs (as of 2023)
    Tahereh Mohammadian Gol, Fatemeh Zahedipour, Paul Trosien, Guillermo Ureña-Bailén, Miso Kim, Justin S. Antony, Markus Mezger · 2024 · Life Sciences
53. Parental perspectives on gene therapy for children with haemophilia: The Exigency study
    Kate Khair, Lisa Steadman, Steve Chaplin · 2020 · Haemophilia
54. Management of adrenoleukodystrophy: From pre-clinical studies to the development of new therapies
    Chui Yan Ma, Cheng Li, Xiaoya Zhou, Zhao Zhang, Hua Jiang, Hongsheng Liu, Huanhuan Joyce Chen, Hung‐Fat Tse, Can Liao, Qizhou Lian · 2021 · Biomedicine & Pharmacotherapy
55. IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy
    Qiushi Liang, Fabio Catalano, Eva C. Vlaar, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Ans T. van der Ploeg, Niek Peter van Til, W.W.M. Pim Pijnappel · 2022 · Molecular Therapy — Methods & Clinical Development
56. Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach
    Laura Ann Adang, Anjana Sevagamoorthy, Omar Sherbini, Jamie L. Fraser, Joshua L. Bonkowsky, Francesco Gavazzi, Russel D'Aiello, Nicholson B. Modesti, Emily J. Yu, Sylvia Mutua et al. · 2024 · Molecular Genetics and Metabolism
57. Natural history of a cohort of <i><scp>ABCD</scp>1</i> variant female carriers
    Schirinzi, T., Vasco, G., Aiello, C., Rizzo, C., Sancesario, A., Romano, A., Favetta, M., Petrarca, M., Paone, L., Castelli, E. et al. · 2019 · European Journal of Neurology
58. Clinical efficacy of haematopoietic stem cell transplantation for adult adrenoleukodystrophy
    Takashi Matsukawa, Tomotaka Yamamoto, Akira Honda, Takashi Toya, Hiroyuki Ishiura, Jun Mitsui, Masaki Tanaka, Akihito Hao, Akihito Shinohara, Mizuki Ogura et al. · 2020 · Brain Communications
59. High‐dose biotin restores redox balance, energy and lipid homeostasis, and axonal health in a model of adrenoleukodystrophy
    Stéphane Fourcade, Leire Goicoechea, Janani Parameswaran, Agatha Schlüter, Nathalie Launay, Montserrat Ruíz, Alexandre Seyer, Benoît Colsch, Noel Ylagan Calingasan, Isidró Ferrer et al. · 2020 · Brain Pathology
60. The transformative potential of HSC gene therapy as a genetic medicine
    Pervinder Sagoo, Hubert Bobby Gaspar · 2021 · Gene Therapy
61. Leukodystrophies in Children: Diagnosis, Care, and Treatment
    Joshua L. Bonkowsky, Stephanie R. Keller, Jamie K. Capal, Timothy Edward Lotze, Renée A. Shellhaas, David K. Urion, Emily Chen, Tracy L. Trotter, Susan A. Berry, Leah W. Burke et al. · 2021 · PEDIATRICS
62. Molecular Biomarkers for Adrenoleukodystrophy: An Unmet Need
    Madison I. J. Honey, Yorrick R. J. Jaspers, Marc Engelen, Stephan Kemp, Irene C. Huffnagel · 2021 · Cells
63. Stem cell-derived CAR T cells show greater persistence, trafficking, and viral control compared to ex vivo transduced CAR T cells
    Mayra A. Carrillo, Anjie Zhen, Wenli Mu, Valerie K. Rezek, Heather A. Martin, Christopher W. Peterson, Hans‐Peter Kiem, Scott G. Kitchen · 2024 · Molecular Therapy
64. New Methods for Disease Modeling Using Lentiviral Vectors
    Arántzazu Alfranca, Miguel R. Campanero, Juan Miguel Redondo · 2018 · Trends in Molecular Medicine
65. Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases
    Jacob Favret, Nadav I. Weinstock, M. Laura Feltri, Daesung Shin · 2020 · Frontiers in Molecular Biosciences
66. In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment
    Sung-Ah Hong, Jung Hwa Seo, Soohyun Wi, Eul Sik Jung, Jihyeon Yu, Gue‐Ho Hwang, Ji Hea Yu, Ahreum Baek, Soeon Park, Sangsu Bae et al. · 2021 · Molecular Therapy
67. Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy
    Lauer, Arne, Speroni, Samantha L., Choi, Myoung, Da, Xiao, Duncan, Christine, McCarthy, Siobhan, Krishnan, Vijai, Lusk, Cole A., Rohde, David, Hansen, Mikkel Bo et al. · 2023 · Nature Communications
68. Potential use of stem cells as a therapy for cystinosis
    Céline J. Rocca, Stéphanie Cherqui · 2018 · Pediatric Nephrology
69. Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches
    Andrés Lamsfus‐Calle, Alberto Daniel-Moreno, Guillermo Ureña-Bailén, Janani Raju, Justin S. Antony, Rupert Handgretinger, Markus Mezger · 2019 · Blood Reviews
70. Immunoresponse to Gene-Modified Hematopoietic Stem Cells
    C. Drysdale, J. Tisdale, N. Uchida · 2019 · Molecular Therapy: Methods & Clinical Development
71. Allogeneic HSCT transfers wild-type cystinosin to nonhematological epithelial cells in cystinosis: First human report
    Mohamed A. Elmonem, Koenraad R. P. Veys, Fanny Oliveira Arcolino · 2018 · American Journal of Transplantation
72. Diagnostic approach in adult-onset neurometabolic diseases
    Gorka Fernández‐Eulate, Christophe Carreau, Jean‐François Benoist, Foudil Lamari, Benoît Rucheton, Natalia Shor, Yann Nadjar · 2022 · Journal of Neurology Neurosurgery & Psychiatry
73. Hematopoietic Stem Cell Transplantation for Neurological Disorders: A Focus on Inborn Errors of Metabolism
    Pedro Vasconcelos, João Forjaz de Lacerda · 2022 · Frontiers in Cellular Neuroscience
74. Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy
    Serena Scala, Francesca Ferrua, Luca Basso Ricci, Francesca Dionisio, Maryam Omrani, Pamela Quaranta, Raisa Jofra Hernández, Luca Del Core, Fabrizio Benedicenti, Ilaria Monti et al. · 2023 · Nature Communications
75. Neurosurgical gene therapy for central nervous system diseases
    Ruchit V. Patel, Pranav Nanda, Robert Mark Richardson · 2024 · Neurotherapeutics
76. Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia
    M. Lidonnici, Y. Paleari, F. Tiboni, G. Mandelli, C. Rossi, M. Vezzoli, A. Aprile, C. Lederer, A. Ambrosi, F. Chanut et al. · 2018 · Molecular Therapy: Methods & Clinical Development
77. Hematopoietic Stem Cell Gene Therapy for Brain Metastases Using Myeloid Cell–Specific Gene Promoters
    Tereza Andreou, Nora Rippaus, Krzysztof Wronski, Jennifer M. Williams, David Taggart, Stéphanie Cherqui, Ashley Sunderland, Yolanda Dyah Kartika, Teklu Egnuni, Rebecca J. Brownlie et al. · 2019 · JNCI Journal of the National Cancer Institute
78. Vorinostat in the acute neuroinflammatory form of X‐linked adrenoleukodystrophy
    Bettina Zierfuss, Isabelle Weinhofer, Jörn‐Sven Kühl, Wolfgang Köhler, Annette E. Bley, Katharina Zauner, Johannes Binder, Ksenija Martinović, Christian Seiser, Christoph Hertzberg et al. · 2020 · Annals of Clinical and Translational Neurology
79. Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases
    Estera Rintz, Takashi Higuchi, Hiroshi Kobayashi, Deni S. Galileo, Grzegorz Węgrzyn, Shunji Tomatsu · 2021 · Molecular Therapy — Methods & Clinical Development
80. AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency
    Xiao-Hui Liao, Pablo Avalos, Oksana Shelest, Raz Ofan, Michael Shilo, Catherine J. Bresee, Shibi B. Likhite, Jean‐Philippe Vit, Heike Heuer, Brian K. Kaspar et al. · 2022 · Thyroid
81. The peroxisomal fatty acid transporter ABCD1/PMP-4 is required in the C. elegans hypodermis for axonal maintenance: A worm model for adrenoleukodystrophy
    Coppa, Andrea, Guha, Sanjib, Fourcade, Stéphane, Parameswaran, Janani, Ruiz, Montserrat, Moser, Ann B., Schlüter, Agatha, Murphy, Michael P., Lizcano, Jose Miguel, Miranda-Vizuete, Antonio et al. · 2020 · Free Radical Biology and Medicine
82. Viral gene therapy for paediatric neurological diseases: progress to clinical reality
    R. Privolizzi, W. Chu, Maha Tijani, J. Ng · 2021 · Developmental Medicine & Child Neurology
83. Genetically modified macrophages accelerate myelin repair
    Marie‐Stéphane Aigrot, Clara Barthélemy, Sarah Moyon, Gaelle Dufayet‐Chaffaud, Leire Izagirre‐Urizar, Béatrix Gillet-Legrand, Laura Bayón‐Cordero, Satoru Tada, Juan Carlos Chara, Carlos Matute et al. · 2022 · EMBO Molecular Medicine
84. Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier
    Bethan J. Critchley, Hubert Bobby Gaspar, Sara Benedetti · 2022 · Molecular Therapy
85. Newborn Screening and Emerging Therapies for X-Linked Adrenoleukodystrophy
    Moser, Ann B., Fatemi, Ali · 2018 · JAMA Neurology
86. Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1
    Raymond Hickey, Clara T. Nicolas, Kari L. Allen, Shennen A. Mao, Faysal Elgilani, Jaime M. Glorioso, Bruce P. Amiot, Caitlin J. VanLith, Rebekah M. Guthman, Zeji Du et al. · 2018 · Cell Transplantation
87. Current Advances and Challenges in Gene Therapies for Neurologic Disorders
    Giulia S. Porcari, John Warren Collyer, Laura Ann Adang, Deepa Soundara Rajan · 2025 · Neurology Genetics
88. Newborn Screening for X-Linked Adrenoleukodystrophy in Nebraska: Initial Experiences and Challenges
    Craig V. Baker, Alyssa Cady Keller, Richard Lutz, Karen Eveans, Krystal Baumert, James C. DiPerna, William B. Rizzo · 2022 · International Journal of Neonatal Screening
89. Gene therapies for mucopolysaccharidoses
    Alessandro Rossi, Nicola Brunetti‐Pierri · 2023 · Journal of Inherited Metabolic Disease
90. Stem cell heterogeneity, plasticity, and regulation
    Ziyang Cui, Hope Wei, Colin R. Goding, Rutao Cui · 2023 · Life Sciences
91. Biogenesis and Function of Peroxisomes in Human Disease with a Focus on the ABC Transporter
    Tsuneo Imanaka · 2019 · Biological and Pharmaceutical Bulletin
92. Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
    Maria Dahl, Emma M. K. Smith, Sarah Warsi, Michael Rothe, Maria J. Ferraz, Johannes M. F. G. Aerts, Azadeh Golipour, Claudia G. Harper, Richard W. Pfeifer, Daniella M. Pizzurro et al. · 2020 · Molecular Therapy — Methods & Clinical Development
93. Clinical hematopoietic stem cell-based gene therapy
    Tami John, A. Czechowicz · 2025 · Molecular Therapy
94. Reducing Suppressors of Cytokine Signaling-3 (SOCS3) Expression Promotes M2 Macrophage Polarization and Functional Recovery After Intracerebral Hemorrhage
    Xinchao Ji, Ya-Jun Shi, Yan Zhang, Mingze Chang, Gang Zhao · 2020 · Frontiers in Neurology
95. POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches
    Stefanie Perrier, Mackenzie A. Michell‐Robinson, Geneviève Bernard · 2021 · Frontiers in Cellular Neuroscience
96. Evaluation of Neurofilament Light Chain as a Biomarker of Neurodegeneration in X-Linked Childhood Cerebral Adrenoleukodystrophy
    Hongge Wang, Matthew D. Davison, Martin L. Kramer, Weiliang Qiu, Tatiana B. Gladysheva, Ruby M. S. Chiang, Can Kayatekin, David R. Nascene, Leyla A. Taghizadeh, Carina J. King et al. · 2022 · Cells
97. The Role of Oxidative Stress and Inflammation in X-Link Adrenoleukodystrophy
    Jiayu Yu, Ting Chen, Xin Guo, Mohammad Ishraq Zafar, Huiqing Li, Zhihua Wang, Juan Zheng · 2022 · Frontiers in Nutrition
98. Genetic testing in adults with neurologic disorders: indications, approach, and clinical impacts
    Laynie Dratch, Meron Azage, Aaron Baldwin, Kelsey Johnson, Rachel A Paul, Tanya Bardakjian, Sara-Claude Michon, Defne A. Amado, Michael Baer, Andres Deik et al. · 2023 · Journal of Neurology
99. Reduced-Toxicity (BuFlu) Conditioning Is Better Tolerated but Has a Higher Second Transplantation Rate Compared to Myeloablative Conditioning (BuCy) in Children with Inherited Metabolic Disorders
    Ashish O. Gupta, Michael C. Downey, Ryan Shanley, Cathryn A. Jennissen, Weston P. Miller, Troy C. Lund, Paul J. Orchard, Angela R. Smith · 2019 · Transplantation and Cellular Therapy
100. Geographic and Specialty Access Disparities in US Pediatric Leukodystrophy Diagnosis
     Sara Elizabeth Grineski, Danielle Xiaodan Morales, Timothy W. Collins, Jacob Wilkes, Joshua L. Bonkowsky · 2020 · The Journal of Pediatrics