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Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome

Kuo, Caroline Y. and Hoban, Megan D. and Joglekar, Alok V. and Young, Courtney S. and Kleinman, John and Abele, Alison N. and Kohn, Donald B. (2015) Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome. Molecular Therapy, 23 (S1). S50. ISSN 1525-0016. https://resolver.caltech.edu/CaltechAUTHORS:20150914-153726915

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Abstract

X-linked hyper-IgM syndrome (XHIM) is a primary immunodeficiency due to defects in immunoglobulin class switch recombination caused by mutations in the CD40 ligand (CD40L) gene. Previous gene therapy-based studies have investigated the use of retroviral vectors for delivery of CD40L cDNA. Due to uncontrolled expression of the gene, this method resulted in significant abnormal lymphoproliferation in mouse models, emphasizing the need for alternative strategies. Site-specific genome modification provides the promise for correction of the gene under the regulation of its endogenous enhancer and promoter.


Item Type:Article
Related URLs:
URLURL TypeDescription
http://dx.doi.org/10.1038/mt.2015.74DOIAbstracts
http://www.nature.com/mt/journal/v23/n1s/full/mt201574a.htmlPublisherAbstracts
ORCID:
AuthorORCID
Joglekar, Alok V.0000-0001-7554-7447
Additional Information:© 2015 American Society of Gene & Cell Therapy.
Issue or Number:S1
Record Number:CaltechAUTHORS:20150914-153726915
Persistent URL:https://resolver.caltech.edu/CaltechAUTHORS:20150914-153726915
Usage Policy:No commercial reproduction, distribution, display or performance rights in this work are provided.
ID Code:60233
Collection:CaltechAUTHORS
Deposited By: Tony Diaz
Deposited On:14 Sep 2015 23:09
Last Modified:09 Mar 2020 13:18

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