254. Correction of RAG2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Requires Strong Expression of RAG2

Molecular Therapy ◽

10.1016/s1525-0016(16)36826-5 ◽

2011 ◽

Vol 19 ◽

pp. S99

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Strong Expression ◽

Lentiviral Gene Therapy

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Faculty Opinions recommendation of Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene.

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

10.3410/f.722644269.793515036 ◽

2016 ◽

Author(s):

Fabio Candotti

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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617. Lentiviral Gene Therapy of RAG1 Severe Combined Immunodeficiency: Risks and Implications for Treatment

Molecular Therapy ◽

10.1016/s1525-0016(16)38058-3 ◽

2010 ◽

Vol 18 ◽

pp. S240

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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230. Correction of Immunity in Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-Optimized RAG2 Therapeutic Transgene

Molecular Therapy ◽

10.1016/s1525-0016(16)36034-8 ◽

2012 ◽

Vol 20 ◽

pp. S90-S91

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene

Molecular Therapy ◽

10.1038/mt.2012.110 ◽

2012 ◽

Vol 20 (10) ◽

pp. 1968-1980 ◽

Author(s):

Niek P van Til ◽

Helen de Boer ◽

Nomusa Mashamba ◽

Agnieszka Wabik ◽

Marshall Huston ◽

...

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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243. Pre-Clinical Development of Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency

Molecular Therapy ◽

10.1016/s1525-0016(16)33848-5 ◽

2015 ◽

Vol 23 ◽

pp. S95

Author(s):

Valentina Poletti ◽

Sabine Charrier ◽

Samia Martin ◽

Arianna Moiani ◽

Céline Sagrère ◽

...

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Clinical Development ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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661. Lentiviral Gene Therapy of RAG Severe Combined Immunodeficiency

Molecular Therapy ◽

10.1016/s1525-0016(16)34996-6 ◽

2013 ◽

Vol 21 ◽

pp. S252 ◽

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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415. Development of a Lentiviral Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency (ADA-SCID)

Molecular Therapy ◽

10.1016/s1525-0016(16)36219-0 ◽

2012 ◽

Vol 20 ◽

pp. S162

Keyword(s):

Gene Therapy ◽

Adenosine Deaminase ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Lentiviral Gene Therapy

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Faculty Opinions recommendation of Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome.

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

10.3410/f.718206925.793515037 ◽

2016 ◽

Author(s):

Fabio Candotti

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Recombination Activating Gene ◽

Lentiviral Gene Therapy ◽

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Recombination-activating gene 1 (Rag1)–deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

Journal of Allergy and Clinical Immunology ◽

10.1016/j.jaci.2013.10.009 ◽

2014 ◽

Vol 133 (4) ◽

pp. 1116-1123 ◽

Author(s):

Niek P. van Til ◽

Roya Sarwari ◽

Trudi P. Visser ◽

Julia Hauer ◽

Chantal Lagresle-Peyrou ◽

...

Keyword(s):

Gene Therapy ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Recombination Activating Gene ◽

Lentiviral Gene Therapy ◽

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Faculty Opinions recommendation of Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

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

10.3410/f.1005837.70356 ◽

2002 ◽

Author(s):

Seppo Yla-Herttuala

Keyword(s):

Gene Therapy ◽

Ex Vivo ◽

Severe Combined Immunodeficiency ◽

Combined Immunodeficiency ◽

Ex Vivo Gene Therapy

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