Search Result "congenital amaurosis."


Manufacturing and Regulatory Strategies for Clinical AAV2-hRPE65

Journal: Current Gene Therapy
Volume: 10 Issue: 5 Year: 2010 Page: 341-349
Author(s): J. Fraser Wright, Jennifer Wellman, Katherine A. High

AAV-Mediated Gene Supply for Treatment of Degenerative and Neovascular Retinal Diseases

Journal: Current Gene Therapy
Volume: 10 Issue: 5 Year: 2010 Page: 371-380
Author(s): Pasqualina Colella, Alberto Auricchio

Review Article

Ocular Ciliopathies: Genetic and Mechanistic Insights into Developing Therapies

Journal: Current Medicinal Chemistry
Volume: 26 Issue: 17 Year: 2019 Page: 3120-3131
Author(s): Mahesh Shivanna,Manisha Anand,Subhabrata Chakrabarti,Hemant Khanna

General Research Article

Leukemia Inhibitory Factor Protects against Degeneration of ConePhotoreceptors Caused by RPE65 Deficiency

Journal: Current Medicinal Chemistry
Volume: 31 Issue: 25 Year: 2024 Page: 4022-4033
Author(s):

Zebrafish Models of Photoreceptor Ciliopathies

Ebook: Molecular Bases of Neurodegenerative Disorders of the Retina
Volume: 1 Year: 2017
Author(s): Brian D. Perkins
Doi: 10.2174/9781681085487117010004

Review Article

The Role of Gene Therapy in the Treatment of Retinal Diseases: A Review

Journal: Current Gene Therapy
Volume: 17 Issue: 3 Year: 2017 Page: 194-213
Author(s): C. Campa,C. E. Gallenga,E. Bolletta,P. Perri

Review Article

Delivering the "Blueprints" or "DNA Repairing Kits" Instead of Drugs in the Treatment of Congenital Hemoglobinopathies

Journal: Current Organic Chemistry
Volume: 21 Issue: 1 Year: 2017 Page: 74-85
Author(s): Diana Chiru,Cristina Mambet,Lilia Matei,Coralia Bleotu,Simona Ruta,Carmen C. Diaconu

Present and Future of Adeno Associated Virus Based Gene Therapy Approaches

Journal: Recent Patents on Endocrine, Metabolic & Immune Drug Discovery
Volume: 6 Issue: 1 Year: 2012 Page: 47-66
Author(s): Saida Ortolano, Carlos Spuch, Carmen Navarro

Current Advances in Gene Therapy for the Treatment of Genodermatoses

Journal: Current Gene Therapy
Volume: 9 Issue: 6 Year: 2009 Page: 487-494
Author(s): Heather A. Long, James R. McMillan, Hongjiang Qiao, Masashi Akiyama, Hiroshi Shimizu

Prospectives for Gene Therapy of Retinal Degenerations

Journal: Current Genomics
Volume: 13 Issue: 5 Year: 2012 Page: 350-362
Author(s): Gabriele Thumann

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