Efficacy and safety of gene therapy with AAV4 in childhood blindness due to rpe65 mutations

2012 ◽  
Vol 90 ◽  
pp. 0-0 ◽  
Author(s):  
G LE MEUR ◽  
P LEBRANCHU ◽  
Y PEREON ◽  
F BILLAUD ◽  
C IVAN ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Efficacy And Safety ◽  
Childhood Blindness

ASPIRO Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Efficacy and Safety Findings

2021 ◽  
Author(s):  
A. Blaschek ◽  
P. B. Shieh ◽  
N. Kuntz ◽  
J. J. Dowling ◽  
W. Müller-Felber ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Efficacy And Safety ◽  
Myotubular Myopathy ◽  
Gene Therapy Trial ◽  
Therapy Trial

scholarly journals Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

2018 ◽  
Vol 8 ◽  
pp. 152-165 ◽  
Author(s):  
Rana Yadak ◽  
Raquel Cabrera-Pérez ◽  
Javier Torres-Torronteras ◽  
Marianna Bugiani ◽  
Joost C. Haeck ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Safety Evaluation ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Preclinical Efficacy ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

2011 ◽  
Vol 19 (3) ◽  
pp. 442-449 ◽  
Author(s):  
Denise E Sabatino ◽  
Amy M Lange ◽  
Ekaterina S Altynova ◽  
Rita Sarkar ◽  
Shangzhen Zhou ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Hemophilia A ◽  
Severe Hemophilia ◽  
Efficacy And Safety ◽  
Liver Gene

scholarly journals Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

2006 ◽  
Vol 5 (1) ◽  
pp. 16-24 ◽  
Author(s):  
T. VANDENDRIESSCHE ◽  
L. THORREZ ◽  
A. ACOSTA-SANCHEZ ◽  
I. PETRUS ◽  
L. WANG ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Lentiviral Vectors ◽  
Hemophilia B ◽  
Efficacy And Safety

Efficacy and Safety of Clinical Grade hVEGF-DΔNΔC Gene Therapy Containing Residual Replication-Competent Adenoviruses

2020 ◽  
Author(s):  
Aleksi Johannes Leikas ◽  
Nihay Laham-Karam ◽  
Eline Agtereek ◽  
Hanna Marjaana Peltonen ◽  
Tuomas Selander ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Grade ◽  
Efficacy And Safety

scholarly journals Preclinical efficacy and safety of KCNH2-G628S gene therapy for postoperative atrial fibrillation

2017 ◽  
Vol 154 (5) ◽  
pp. 1644-1651.e8 ◽  
Author(s):  
Zhao Liu ◽  
Julie A. Hutt ◽  
Barur Rajeshkumar ◽  
Yoshihiro Azuma ◽  
Kailai L. Duan ◽  
...  
Keyword(s):  
Atrial Fibrillation ◽  
Gene Therapy ◽  
Postoperative Atrial Fibrillation ◽  
Efficacy And Safety ◽  
Preclinical Efficacy
Sign in / Sign up

Export Citation Format

Share Document