Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis
Author:
Publisher
Informa Healthcare
Subject
Clinical Biochemistry,Drug Discovery,Pharmacology
Link
http://www.tandfonline.com/doi/pdf/10.1517/14712598.2011.557358
Reference34 articles.
1. Leber Congenital Amaurosis–A Model for Efficient Genetic Testing of Heterogeneous Disorders: LXIV Edward Jackson Memorial Lecture
2. Leber congenital amaurosis: Genes, proteins and disease mechanisms
3. Ocular Gene Therapy: An Evaluation of Recombinant Adeno-Associated Virus-Mediated Gene Therapy Interventions for the Treatment of Ocular Disease
4. In Utero Gene Therapy Rescues Vision in a Murine Model of Congenital Blindness
5. Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy
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4. Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants;Human Gene Therapy;2018-04
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