Fanconi anemia and βc deficiency-associated pulmonary alveolar proteinosis as two hereditary diseases of childhood which are potentially curable by stem cell gene therapy but require different therapeutic approaches

1999 ◽  
Vol 211 (04) ◽  
pp. 329-335 ◽  
Author(s):  
U. Dirksen ◽  
T. Moritz ◽  
S. Burdach ◽  
M. Flasshove ◽  
H. Hanenberg
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Fanconi Anemia ◽  
Pulmonary Alveolar Proteinosis ◽  
Hereditary Diseases ◽  
Therapeutic Approaches ◽  
Alveolar Proteinosis ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting

2011 ◽  
Vol 19 (7) ◽  
pp. 1193-1198 ◽  
Author(s):  
Jakub Tolar ◽  
Jennifer E Adair ◽  
Michael Antoniou ◽  
Cynthia C Bartholomae ◽  
Pamela S Becker ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Fanconi Anemia ◽  
Working Group ◽  
Group Meeting ◽  
Fanconi Anemia Gene ◽  
Cell Gene ◽  
Working Group Meeting ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Stem Cell Gene Therapy for Chronic Renal Failure

2003 ◽  
Vol 3 (5) ◽  
pp. 387-394 ◽  
Author(s):  
T. Yokoo ◽  
K. Sakurai ◽  
T. Ohashi ◽  
T. Kawamura
Keyword(s):  
Gene Therapy ◽  
Renal Failure ◽  
Stem Cell ◽  
Chronic Renal Failure ◽  
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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