Identification of Genes Causing Autosomal Recessive Retinitis Pigmentosa

Mehmood, Rashid; Ramzan, Muhammad; Ali, Akhtar; Riaz, Assad; Zulfiqar, Fareeha; Riazuddin, Sheikh

doi:10.1016/b978-044452806-3/50003-2

Gene Therapy 2006

DOI: 10.1016/b978-044452806-3/50003-2

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Identification of Genes Causing Autosomal Recessive Retinitis Pigmentosa

Rashid Mehmood

Muhammad Ramzan

Akhtar Ali

et al.

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2021

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“…Recent clinical trials for retinal dystrophies arranged by condition and therapeutic approach[21][22][23][24][25][26][27][28][29][30][31][32][33][34][35][36][37].…”

mentioning

confidence: 99%

The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease

Velázquez

Ballios

2021

IJMS

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Inherited retinal degenerations (IRDs) are a diverse group of conditions that are often characterized by the loss of photoreceptors and blindness. Recent innovations in molecular biology and genomics have allowed us to identify the causative defects behind these dystrophies and to design therapeutics that target specific mechanisms of retinal disease. Recently, the FDA approved the first in vivo gene therapy for one of these hereditary blinding conditions. Current clinical trials are exploring new therapies that could provide treatment for a growing number of retinal dystrophies. While the field has had early success with gene augmentation strategies for treating retinal disease based on loss-of-function mutations, many novel approaches hold the promise of offering therapies that span the full spectrum of causative mutations and mechanisms. Here, we provide a comprehensive review of the approaches currently in development including a discussion of retinal neuroprotection, gene therapies (gene augmentation, gene editing, RNA modification, optogenetics), and regenerative stem or precursor cell-based therapies. Our review focuses on technologies that are being developed for clinical translation or are in active clinical trials and discusses the advantages and limitations for each approach.

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“…Recent clinical trials for retinal dystrophies arranged by condition and therapeutic approach[21][22][23][24][25][26][27][28][29][30][31][32][33][34][35][36][37].…”

mentioning

confidence: 99%

The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease

Velázquez

Ballios

2021

IJMS

View full text Add to dashboard Cite

show abstract

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Identification of Genes Causing Autosomal Recessive Retinitis Pigmentosa

Cited by 1 publication

References 29 publications

The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease

The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease

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