Cell and gene therapy critical starting material: a discussion to help establish release specifications for plasmids and the bacterial master cell banks used to produce them

2020 ◽  
Author(s):  
Keyword(s):  
Gene Therapy ◽  
Starting Material ◽  
Cell And Gene Therapy ◽  
Cell Banks

Attenuating Muscle Wasting: Cell and Gene Therapy Approaches

2003 ◽  
Vol 4 (7) ◽  
pp. 575-585 ◽  
Author(s):  
A. Musaro ◽  
N. Rosenthal
Keyword(s):  
Gene Therapy ◽  
Muscle Wasting ◽  
Cell And Gene Therapy

Fetal stem cell and gene therapy

2017 ◽  
Vol 22 (6) ◽  
pp. 410-414 ◽  
Author(s):  
Russell Witt ◽  
Tippi C. MacKenzie ◽  
William H. Peranteau
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Fetal Stem Cell ◽  
Cell And Gene Therapy

How institutional biosafety committees contribute to safety, capacity and regulatory approvals in cell and gene therapy trials

Cytotherapy ◽  
2021 ◽  
Vol 23 (5) ◽  
pp. S206
Author(s):  
G. O’Sullivan ◽  
B. Yu ◽  
C. Bailey ◽  
Z. Velickovic ◽  
J. Rasko
Keyword(s):  
Gene Therapy ◽  
Cell And Gene Therapy

Retinal Transplantation, Stem Cell and Gene Therapy in Retinitis Pigmentosa

2021 ◽  
pp. 131-139
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Retinitis Pigmentosa ◽  
Retinal Dystrophy ◽  
Gene Replacement ◽  
Disease Genes ◽  
Functional Roles ◽  
Tunnel Vision ◽  
Hereditary Retinal Dystrophy ◽  
Cell And Gene Therapy

Retinitis pigmentosa is the most common hereditary retinal dystrophy which has marked clinical and genetic heterogeneity. Common presentations among this disorder include night blindness, tunnel vision, and subsequent progression to complete blindness respectively. The known causative disease genes have a variety of developmental and functional roles, with mutations in more than 120 genes shown to be responsible for the phenotypes. In addition, mutations within the same gene have been shown to cause different disease phenotypes, even within the same family, highlighting further levels of complexity. In recent years significant advancements have been made in the understanding of the pathogenesis of the disease and stem cell and gene replacement treatments have been proposed as potentially efficacious therapies. This review summarizes the clinical development of retinal stem cell and gene therapy.

Proceedings of the first academic symposium on developing, qualifying and operating a cell and gene therapy manufacturing facility

Cytotherapy ◽  
2018 ◽  
Vol 20 (12) ◽  
pp. 1486-1494 ◽  
Author(s):  
DAVID L. DIGIUSTO ◽  
KATHRYN MELSOP ◽  
RASHI SRIVASTAVA ◽  
CHY-ANH T. TRAN
Keyword(s):  
Gene Therapy ◽  
Manufacturing Facility ◽  
A Cell ◽  
Cell And Gene Therapy
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