Hematopoietic stem cell (HSC) gene therapy for two patients with adenosine deaminase (ADA) deficiency without cytoreductive conditioning; a suggestion for the optimal protocol for HSC gene therapy for ADA deficiency

2008 ◽  
Vol 40 (2) ◽  
pp. 251
Author(s):  
Tadashi Ariga
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Adenosine Deaminase ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Optimal Protocol

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID

2009 ◽  
Vol 44 (1-3) ◽  
pp. 150-159 ◽  
Author(s):  
Alessandro Aiuti ◽  
Immacolata Brigida ◽  
Francesca Ferrua ◽  
Barbara Cappelli ◽  
Robert Chiesa ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Adenosine Deaminase ◽  
Hematopoietic Stem Cell ◽  
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

Sickle Cell Disease Hematopoietic Stem Cell gene therapy with globin gene addition is promising

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Sickle Cell Disease ◽  
Hematopoietic Stem Cell ◽  
Sickle Cell ◽  
Globin Gene ◽  
Autologous Transplantation ◽  
Gene Repair ◽  
Cell Disease ◽  
Hematopoietic Stem

Autologous transplantation of gene-modified HSCs might be used to treat Sickle Cell Disease (SCD) once and for all. Hematopoietic Stem Cell (HSC) gene therapy with lentiviral-globin gene addition was optimized by HSC collection, vector constructs, lentiviral transduction, and conditioning in the current gene therapy experiment for SCD, resulting in higher gene marking and phenotypic correction. Further advancements over the next decade should allow for a widely approved gene-addition therapy. Long-term engraftment is crucial for gene-corrected CD34+ HSCs, which might be addressed in the coming years, and gene repair of the SCD mutation in the-globin gene can be achieved in vitro using genome editing in CD34+ cells. Because of breakthroughs in efficacy, safety, and delivery strategies, in vivo gene addition and gene correction in BM HSCs is advancing. Overall, further research is needed, but HSC-targeted gene addition/gene editing therapy is a promising SCD therapy with curative potential that might be widely available soon.

scholarly journals Development of a modified selective amplifier gene for hematopoietic stem cell gene therapy

Gene Therapy ◽  
1999 ◽  
Vol 6 (6) ◽  
pp. 1038-1044 ◽  
Author(s):  
K M Matsuda ◽  
A Kume ◽  
Y Ueda ◽  
M Urabe ◽  
M Hasegawa ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Selective Amplifier ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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