Faculty Opinions recommendation of Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.

2016 ◽  
Author(s):  
Fabio Candotti
Keyword(s):  
Gene Therapy ◽  
Severe Combined Immunodeficiency ◽  
Combined Immunodeficiency ◽  
Hematopoietic Stem ◽  
Immunological Recovery ◽  
Metabolic Correction ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

2016 ◽  
Vol 8 (335) ◽  
pp. 335ra57-335ra57 ◽  
Author(s):  
Suk See De Ravin ◽  
Xiaolin Wu ◽  
Susan Moir ◽  
Lela Kardava ◽  
Sandra Anaya-O’Brien ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Severe Combined Immunodeficiency ◽  
Combined Immunodeficiency ◽  
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

Long-term efficacy and safety of hematopoietic stem cell gene therapy mediated by lentiviral vectors in the murine model of Wiskott-Aldrich syndrome

2008 ◽  
Vol 40 (2) ◽  
pp. 256-257
Author(s):  
Marita Bosticardo ◽  
Francesco Marangoni ◽  
Michela Locci ◽  
Elena Draghici ◽  
Samantha Scaramuzza ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Lymphoid Development and Function in X-Linked Severe Combined Immunodeficiency Mice after Stem Cell Gene Therapy

2000 ◽  
Vol 1 (2) ◽  
pp. 145-153 ◽  
Author(s):  
Makoto Otsu ◽  
Stacie M. Anderson ◽  
David M. Bodine ◽  
Jennifer M. Puck ◽  
John J. O'Shea ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Severe Combined Immunodeficiency ◽  
Combined Immunodeficiency ◽  
Lymphoid Development ◽  
And Function ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Long-term follow-up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

2021 ◽  
Author(s):  
Pierre BOUGNERES ◽  
Salima Hacein-Bey-Abina ◽  
Ivan Labik ◽  
Catherine ADAMSBAUM ◽  
CLEMENCE CASTAIGNEDE ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Long Term Follow Up ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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