Strategies for Treating Inherited Retinal Degeneration With Large Genes That Are Not Amenable to Adeno-Associated Virus–Based Gene Replacement Therapy

2021 ◽  
Author(s):  
Paul Yang ◽  
Mark E. Pennesi ◽  
Richard G. Weleber
Keyword(s):  
Replacement Therapy ◽  
Retinal Degeneration ◽  
Gene Replacement ◽  
Adeno Associated Virus ◽  
Gene Replacement Therapy ◽  
Large Genes ◽  
Inherited Retinal Degeneration

scholarly journals A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

2015 ◽  
Vol 24 (14) ◽  
pp. 3956-3970 ◽  
Author(s):  
Zhijian Wu ◽  
Suja Hiriyanna ◽  
Haohua Qian ◽  
Suddhasil Mookherjee ◽  
Maria M. Campos ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Retinal Degeneration ◽  
Gene Replacement ◽  
Efficacy Study ◽  
Term Efficacy ◽  
Gene Replacement Therapy

scholarly journals In Contrast to AAV-Mediated Cntf Expression, AAV-Mediated Gdnf Expression Enhances Gene Replacement Therapy in Rodent Models of Retinal Degeneration

2006 ◽  
Vol 14 (5) ◽  
pp. 700-709 ◽  
Author(s):  
Prateek K. Buch ◽  
Robert E. MacLaren ◽  
Yanaí Durán ◽  
Kamaljit S. Balaggan ◽  
Angus MacNeil ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Retinal Degeneration ◽  
Gene Replacement ◽  
Rodent Models ◽  
Gene Replacement Therapy

scholarly journals MEN1 Gene Replacement Therapy Reduces Proliferation Rates in a Mouse Model of Pituitary Adenomas

2012 ◽  
Vol 72 (19) ◽  
pp. 5060-5068 ◽  
Author(s):  
Gerard V. Walls ◽  
Manuel C. Lemos ◽  
Mahsa Javid ◽  
Miriam Bazan-Peregrino ◽  
Jeshmi Jeyabalan ◽  
...  
Keyword(s):  
Mouse Model ◽  
Replacement Therapy ◽  
Pituitary Adenomas ◽  
Gene Replacement ◽  
Men1 Gene ◽  
Gene Replacement Therapy

Delivery of cytosolic liver arginase into the mitochondrial matrix space: A possible novel site for gene replacement therapy

1996 ◽  
Vol 22 (6) ◽  
pp. 489-498 ◽  
Author(s):  
Paul B. Wissmann ◽  
Barbara K. Goodman ◽  
Joseph G. Vockley ◽  
Rita M. Kern ◽  
Stephen D. Cederbaum ◽  
...  
Keyword(s):  
Replacement Therapy ◽  
Gene Replacement ◽  
Mitochondrial Matrix ◽  
Matrix Space ◽  
Gene Replacement Therapy
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