Gene therapy for neurodegenerative disorders in children: dreams and realities
Author:
Funder
Pfizer
Novartis
Publisher
Elsevier BV
Subject
Pediatrics, Perinatology and Child Health
Reference54 articles.
1. Editorial: Gene Therapy in the CNS – Progress and Prospects for Novel Therapies;Ledri;Front Mol Neurosci,2021
2. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future;Uddin;Front Oncol,2020
3. Gene specific therapies - The next therapeutic milestone in neurology;Brenner;Neurol Res Pract,2020
4. Van Hout C V. Statistical approaches to rare disease analyses. Genomics of Rare Diseases: Understanding Disease Genetics Using Genomic Approaches, 2021. https://doi.org/10.1016/B978-0-12-820140-4.00011-9.
5. AAV-based in vivo gene therapy for neurological disorders;Ling;Nat Rev Drug Discov,2023
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