ASPIRO Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Efficacy and Safety Findings

Mapping Intimacies ◽

10.1055/s-0041-1739645 ◽

2021 ◽

Author(s):

A. Blaschek ◽

P. B. Shieh ◽

N. Kuntz ◽

J. J. Dowling ◽

W. Müller-Felber ◽

...

Keyword(s):

Gene Therapy ◽

Efficacy And Safety ◽

Myotubular Myopathy ◽

Gene Therapy Trial ◽

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Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

Genetic Engineering & Biotechnology News ◽

10.1089/gen.40.08.05 ◽

2020 ◽

Vol 40 (8) ◽

pp. 14, 16

Author(s):

James M. Wilson ◽

Terence R. Flotte

Keyword(s):

Gene Therapy ◽

Myotubular Myopathy ◽

Gene Therapy Trial ◽

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Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

Human Gene Therapy ◽

10.1089/hum.2020.182 ◽

2020 ◽

Vol 31 (13-14) ◽

pp. 695-696 ◽

Author(s):

James M. Wilson ◽

Terence R. Flotte

Keyword(s):

Gene Therapy ◽

Myotubular Myopathy ◽

Gene Therapy Trial ◽

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Re: “Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy” by Wilson and Flotte

Human Gene Therapy ◽

10.1089/hum.2020.217 ◽

2020 ◽

Vol 31 (15-16) ◽

pp. 787-787 ◽

Author(s):

Perry B. Shieh ◽

Carsten G. Bönnemann ◽

Wolfgang Müller-Felber ◽

Astrid Blaschek ◽

James J. Dowling ◽

...

Keyword(s):

Gene Therapy ◽

Myotubular Myopathy ◽

Gene Therapy Trial ◽

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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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Development of a Gene Therapy Trial for Metastatic Prostate Cancer

10.21236/ada473358 ◽

2006 ◽

Author(s):

Thomas A. Gardner

Keyword(s):

Prostate Cancer ◽

Gene Therapy ◽

Metastatic Prostate Cancer ◽

Gene Therapy Trial ◽

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Development of a Gene Therapy Trial for Metastatic Prostate Cancer

10.21236/ada494544 ◽

2008 ◽

Author(s):

Thomas A. Gardner

Keyword(s):

Prostate Cancer ◽

Gene Therapy ◽

Metastatic Prostate Cancer ◽

Gene 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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Steps in a Translational Cancer Gene Therapy Trial

Cancer Drug Discovery and Development - Gene Therapy for Cancer ◽

10.1007/978-1-59745-222-9_25 ◽

2007 ◽

pp. 447-460

Author(s):

Urs W. von Holzen ◽

Stephen G. Swisher

Keyword(s):

Gene Therapy ◽

Cancer Gene Therapy ◽

Cancer Gene ◽

Gene Therapy Trial ◽

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Popping the Bubble: Promising Results From a Phase I-II Lentiviral Gene Therapy Trial for X-SCID

The Hematologist ◽

10.1182/hem.v16.4.9661 ◽

2019 ◽

Vol 16 (4) ◽

Author(s):

David T. Teachey ◽

Danielle E. Arnold

Keyword(s):

Gene Therapy ◽

Phase I ◽

Gene Therapy Trial ◽

Therapy Trial ◽

Lentiviral Gene Therapy

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