Gene therapy for the treatment of adenosine deaminase-deficient severe combined immune deficiency

Expert Opinion on Orphan Drugs ◽

10.1080/21678707.2017.1325360 ◽

2017 ◽

Vol 5 (6) ◽

pp. 477-485

Author(s):

Robert Sokolic ◽

Fabio Candotti

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Severe Combined Immune Deficiency ◽

Combined Immune Deficiency

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Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2014.11.123 ◽

2015 ◽

Vol 21 (2) ◽

pp. S102

Author(s):

Donald B. Kohn ◽

Kit L. Shaw ◽

Robert Sokolic ◽

Denise A. Carbonaro ◽

Alejandar Davila ◽

...

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Severe Combined Immune Deficiency ◽

Autologous Transplant ◽

Combined Immune Deficiency

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30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector

Molecular Therapy ◽

10.1016/s1525-0016(16)33634-6 ◽

2015 ◽

Vol 23 ◽

pp. S13-S14 ◽

Author(s):

Kit L. Shaw ◽

Elizabeth Garabedian ◽

Rob Sokolic ◽

Provaboti Barman ◽

Alejandra Davila ◽

...

Keyword(s):

Immune Deficiency ◽

Clinical Trial ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Phase Ii ◽

Retroviral Vector ◽

Phase Ii Clinical Trial ◽

Severe Combined Immune Deficiency ◽

Combined Immune Deficiency

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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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Gene therapy techniques in the treatment of adenosine deaminase—deficiency severe combined immune deficiency syndrome

The Journal of Perinatal & Neonatal Nursing ◽

10.1097/00005237-199403000-00007 ◽

1994 ◽

Vol 7 (4) ◽

pp. 65-78 ◽

Author(s):

Nancy A. Noonan ◽

Anne M. Senner

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Immune Deficiency Syndrome ◽

Deficiency Syndrome ◽

Adenosine Deaminase Deficiency ◽

Severe Combined Immune Deficiency ◽

Combined Immune Deficiency

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355. Immune Reconstitution after Gene Therapy for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)

Molecular Therapy ◽

10.1016/s1525-0016(16)38713-5 ◽

2009 ◽

Vol 17 ◽

pp. S138 ◽

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Immune Reconstitution ◽

Severe Combined Immune Deficiency ◽

Combined Immune Deficiency

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68: Gene Therapy for Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Comparative Results with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2007.12.076 ◽

2008 ◽

Vol 14 (2) ◽

pp. 27-28 ◽

Author(s):

G. Podsakoff ◽

B.C. Engel ◽

R. Sokolic ◽

D.A. Carbonaro ◽

L. Muul ◽

...

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Severe Combined Immune Deficiency ◽

Myelosuppressive Chemotherapy ◽

Combined Immune Deficiency ◽

Comparative Results

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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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Immune Reconstitution After Gene Therapy (GTx) for Adenosine Deaminase Deficient Severe Combined Immune Deficiency (ADA-SCID)

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2008.12.081 ◽

2009 ◽

Vol 15 (2) ◽

pp. 27 ◽

Author(s):

R. Sokolic ◽

G. Podsakoff ◽

L. Muul ◽

B. Engel ◽

J. Jagadeesh ◽

...

Keyword(s):

Immune Deficiency ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Immune Reconstitution ◽

Severe Combined Immune Deficiency ◽

Combined Immune Deficiency

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

Molecular Therapy ◽

10.1016/s1525-0016(16)35292-3 ◽

2014 ◽

Vol 22 ◽

pp. S107

Keyword(s):

Immune Deficiency ◽

Clinical Trial ◽

Gene Therapy ◽

Adenosine Deaminase ◽

Phase Ii ◽

Phase Ii Clinical Trial ◽

Severe Combined Immune Deficiency ◽

Combined Immune Deficiency

Download Full-text