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

Jakub Tolar; Jennifer E Adair; Michael Antoniou; Cynthia C Bartholomae; Pamela S Becker; Bruce R Blazar; Juan Bueren; Thomas Carroll; Marina Cavazzana-Calvo; D Wade Clapp; Robert Dalgleish; Anne Galy; H Bobby Gaspar; Helmut Hanenberg; Christof Von Kalle; Hans-Peter Kiem; Dirk Lindeman; Luigi Naldini; Susana Navarro; Raffaele Renella; Paula Rio; Julián Sevilla; Manfred Schmidt; Els Verhoeyen; John E Wagner; David A Williams; Adrian J Thrasher

doi:10.1038/mt.2011.78

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

Molecular Therapy ◽

10.1038/mt.2011.78 ◽

2011 ◽

Vol 19 (7) ◽

pp. 1193-1198 ◽

Cited By ~ 30

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

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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

Klinische Pädiatrie ◽

10.1055/s-2008-1043810 ◽

1999 ◽

Vol 211 (04) ◽

pp. 329-335 ◽

Cited By ~ 4

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

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Anchored Lentiviral Vector Episomes for Stem Cell Gene Therapy in Fanconi Anemia

Current Gene Therapy ◽

10.2174/1566523217666170113165407 ◽

2017 ◽

Vol 16 (5) ◽

pp. 329-337 ◽

Cited By ~ 2

Author(s):

Santhosh Verghese ◽

Peter Kurre

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Fanconi Anemia ◽

Lentiviral Vector ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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A Possible Approach for Stem Cell Gene Therapy of Fanconi Anemia

Current Gene Therapy ◽

10.2174/156652309787354667 ◽

2009 ◽

Vol 9 (1) ◽

pp. 26-32 ◽

Cited By ~ 2

Author(s):

Liting Song

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Fanconi Anemia ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Faculty Opinions recommendation of Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.4242977.4039078 ◽

2010 ◽

Author(s):

Karen E Pollok ◽

Shanbao Cai

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Nonhuman Primate ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Stem Cell Gene Therapy for Chronic Renal Failure

Current Gene Therapy ◽

10.2174/1566523034578221 ◽

2003 ◽

Vol 3 (5) ◽

pp. 387-394 ◽

Cited By ~ 15

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

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

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.263 ◽

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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Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD)

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2017.04.986 ◽

2017 ◽

Vol 21 ◽

pp. e20

Author(s):

F. Fumagalli ◽

V. Calbi ◽

A. Zambon ◽

F. Ciotti ◽

L. Lorioli ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Metachromatic Leukodystrophy ◽

Hematopoietic Stem ◽

Safety And Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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688. Towards a Stem Cell Gene Therapy for Breast Cancer

Molecular Therapy ◽

10.1016/s1525-0016(16)39046-3 ◽

2009 ◽

Vol 17 ◽

pp. S263

Keyword(s):

Breast Cancer ◽

Gene Therapy ◽

Stem Cell ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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276. Immunological and Metabolic Correction After Lentiviral Vector Mediated Haematopoietic Stem Cell Gene Therapy for ADA Deficiency

Molecular Therapy ◽

10.1016/s1525-0016(16)35289-3 ◽

2014 ◽

Vol 22 ◽

pp. S106 ◽

Cited By ~ 4

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Lentiviral Vector ◽

Haematopoietic Stem Cell ◽

Metabolic Correction ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy

Hematology/Oncology Clinics of North America ◽

10.1016/j.hoc.2017.06.006 ◽

2017 ◽

Vol 31 (5) ◽

pp. 721-735 ◽

Cited By ~ 11

Author(s):

Donald B. Kohn

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Historical Perspective ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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