Retinal Dystrophies: Functional Genomics to Gene Therapy

2003 ◽  
Keyword(s):  
Gene Therapy ◽  
Functional Genomics ◽  
Retinal Dystrophies

scholarly journals Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy

2007 ◽  
Vol 8 (Suppl 1) ◽  
pp. S12 ◽  
Author(s):  
Christopher S Hackett ◽  
Aron M Geurts ◽  
Perry B Hackett
Keyword(s):  
Gene Therapy ◽  
Functional Genomics ◽  
Insertion Sites ◽  
Dna Vector

Gene Therapy Tools for Functional Genomics

Genomics ◽  
2002 ◽  
Vol 80 (1) ◽  
pp. 1 ◽  
Author(s):  
Fintan R. Steele
Keyword(s):  
Gene Therapy ◽  
Functional Genomics

scholarly journals Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies

2010 ◽  
Vol 120 (9) ◽  
pp. 3042-3053 ◽  
Author(s):  
Anneke I. den Hollander ◽  
Aaron Black ◽  
Jean Bennett ◽  
Frans P.M. Cremers
Keyword(s):  
Gene Therapy ◽  
Retinal Dystrophies

Surgical Aspects in Gene Therapy for Inherited Retinal Diseases

2021 ◽  
Author(s):  
M. Dominik Fischer ◽  
Karl Ulrich Bartz-Schmidt ◽  
Spyridon Dimopoulos ◽  
Philipp Herrmann ◽  
Maximilian Gerhardt ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Personalised Medicine ◽  
Inherited Retinal Dystrophies ◽  
Retinal Dystrophies ◽  
Open Questions ◽  
Causal Therapy ◽  
Individual Step ◽  
Alternative Approaches ◽  
Surgical Aspects ◽  
Significant Milestone

AbstractInherited retinal dystrophies (IRD) have been studied since their recognition by Franz Donders and Albrecht von Graefe. It nevertheless took 100 years for a causal therapy to take shape in the form of gene therapy: The approval of Voretigen Neparvovec (VN) for the treatment of hereditary retinal dystrophies due to RPE65 mutations was thus a significant milestone – for the era of personalised medicine in general and ophthalmology in particular. The clinical management around gene therapy applications is complex and requires the cooperation of various experts as a multidisciplinary team. This article describes the requirements, challenges, approaches, and open questions regarding the surgical aspects of gene therapy for retinal dystrophies. The first part outlines the standard surgical treatment. Based on this standard, alternative approaches are indicated for each individual step and their value discussed. Knowledge gaps are defined and in the outlook we speculate on future developments.

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