549. High-Level Clustering of Vector Integrations in the German WAS Clinical Gene Therapy Trial

Molecular Therapy ◽

10.1016/s1525-0016(16)36353-5 ◽

2012 ◽

Vol 20 ◽

pp. S212 ◽

Keyword(s):

Gene Therapy ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

Therapy Trial ◽

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Gene therapy for breast cancer. — review of clinical gene therapy trials for breast cancer and mdr1 gene therapy trial in cancer institute hospital

Breast Cancer ◽

10.2325/jbcs.13.8 ◽

2006 ◽

Vol 13 (1) ◽

pp. 8-15 ◽

Author(s):

Shunji Takahashi ◽

Yoshinori Ito ◽

Kiyohiko Hatake ◽

Yoshikazu Sugimoto

Keyword(s):

Breast Cancer ◽

Gene Therapy ◽

Mdr1 Gene ◽

Cancer Institute Hospital ◽

Gene Therapy Trial ◽

Institute Hospital ◽

Cancer Review ◽

Clinical Gene Therapy ◽

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Single-dose AAV9-CLN6 gene transfer stabilizes motor and language function in CLN6-type Batten disease: Interim results from the first clinical gene therapy trial

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.101 ◽

2020 ◽

Vol 129 (2) ◽

pp. S46-S47

Author(s):

Emily de los Reyes ◽

Kathrin Meyer ◽

Lenora Lehwald ◽

Charles Albright ◽

Jeff Castelli ◽

...

Keyword(s):

Gene Therapy ◽

Single Dose ◽

Gene Transfer ◽

Batten Disease ◽

Language Function ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

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296. Update on a Clinical Gene Therapy Trial for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)

Molecular Therapy ◽

10.1016/s1525-0016(16)37737-1 ◽

2010 ◽

Vol 18 ◽

pp. S113

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Severe Combined Immune Deficiency ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

Combined Immune Deficiency ◽

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307. Phase II Clinical Gene Therapy Trial for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID)

Molecular Therapy ◽

10.1016/s1525-0016(16)34642-1 ◽

2013 ◽

Vol 21 ◽

pp. S118

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Phase Ii ◽

Severe Combined Immune Deficiency ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

Combined Immune Deficiency ◽

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343. Molecular Follow-Up of the German WAS Clinical Gene Therapy Trial

Molecular Therapy ◽

10.1016/s1525-0016(16)36915-5 ◽

2011 ◽

Vol 19 ◽

pp. S133

Keyword(s):

Gene Therapy ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

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53. Update on the U.S. Clinical Gene Therapy Trial for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)

Molecular Therapy ◽

10.1016/s1525-0016(16)35857-9 ◽

2012 ◽

Vol 20 ◽

pp. S22

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Severe Combined Immune Deficiency ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

Combined Immune Deficiency ◽

Therapy Trial ◽

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Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial

Blood ◽

10.1182/blood-2002-09-2800 ◽

2002 ◽

Vol 101 (7) ◽

pp. 2563-2569 ◽

Author(s):

L. M. Muul

Keyword(s):

Gene Therapy ◽

Gene Transfer ◽

Adenosine Deaminase ◽

Immune Reaction ◽

Long Term Results ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

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612. Polyclonal Reconstitution of the Hematopoietic System in Two Patients of a WAS Clinical Gene Therapy Trial

Molecular Therapy ◽

10.1016/s1525-0016(16)38053-4 ◽

2010 ◽

Vol 18 ◽

pp. S238

Keyword(s):

Gene Therapy ◽

Hematopoietic System ◽

Gene Therapy Trial ◽

Clinical Gene Therapy ◽

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Design, Baseline Characteristics, and 2-Year Follow-up from The MPS IIIA Natural History Study Used as Control Group in Gene Therapy Trial

10.1055/s-0039-1685419 ◽

2019 ◽

Author(s):

B. Heron ◽

A. Chakrapani ◽

P. Drevot ◽

R. Giugliani ◽

N. Muschol ◽

...

Keyword(s):

Gene Therapy ◽

Natural History ◽

Control Group ◽

Natural History Study ◽

Gene Therapy Trial ◽

Baseline Characteristics ◽

Therapy Trial ◽

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Faculty Opinions recommendation of Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson's disease.

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

10.3410/f.727492597.793530605 ◽

2017 ◽

Author(s):

Laurent Villard

Keyword(s):

Parkinson’S Disease ◽

Gene Therapy ◽

Parkinson's Disease ◽

Gene Therapy Trial ◽

Long Term Follow Up ◽

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