scholarly journals 880. Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy: Clinical Trial Design

2008 ◽  
Vol 16 ◽  
pp. S328-S329
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Stem Cell ◽  
Trial Design ◽  
Metachromatic Leukodystrophy ◽  
Clinical Trial Design ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy

2021 ◽  
Author(s):  
Justin S. Antony ◽  
Alberto Daniel-Moreno ◽  
Andrés Lamsfus-Calle ◽  
Janani Raju ◽  
Merve Kaftancioglu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Metachromatic Leukodystrophy ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy

2012 ◽  
Vol 20 (6) ◽  
pp. 1084-1094 ◽  
Author(s):  
Jacqueline Corrigan-Curay ◽  
Odile Cohen-Haguenauer ◽  
Marina O'Reilly ◽  
Susan R. Ross ◽  
Hung Fan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Trial Design ◽  
Retroviral Vectors ◽  
Gene Correction ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

2021 ◽  
Vol 132 (2) ◽  
pp. S107
Author(s):  
Niek P. van Til ◽  
Yildirim Dogan ◽  
Cecilia Barese ◽  
Zeenath Unnisa ◽  
Swaroopa Guda ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Preclinical Study ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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