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New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals

New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals a a,b Hailiang Hu and Richard A. Gatti Department of Pathology & Laboratory Medicine and Purpose of review Department of Human Genetics, David Geffen School This review is to highlight the most current mutation-targeted therapeutic approaches of Medicine at UCLA, Los Angeles, California, USA and provide insights into new developments for treating primary immunodeficiencies. Correspondence to Richard A. Gatti, MD, Department Recent findings of Pathology and Laboratory Medicine, David Geffen School of Medicine at UCLA, Los Angeles, Significant progress in mutation-targeted treatment was achieved in the past year with CA 90095-1732, USA the identification and characterization of a translational read-through compound, Tel/fax: +1 310 825 7618; e-mail: rgatti@mednet.ucla.edu PTC124. PTC124 demonstrates a new class of nontoxic bioavailable small drugs. Antisense oligonucleotide-mediated techniques such as splicing redirection, exon Current Opinion in Allergy and Clinical Immunology 2008, 8:540–546 skipping, and mismatch repair have been successfully used to correct splicing, frameshift, and missense mutations, respectively. Delivery of antisense oligonucleotides to mammalian cells, including primary leukocytes and neurons, saw great progress during the past year. Recent advances for other approaches to correct frameshift and missense mutations are also considered. Summary Primary immunodeficiencies http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Current Opinion in Allergy and Clinical Immunology Wolters Kluwer Health

New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals

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References (59)

ISSN
1528-4050
eISSN
1473-6322
DOI
10.1097/ACI.0b013e328314b63b
pmid
18978469
Publisher site
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Abstract

New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals a a,b Hailiang Hu and Richard A. Gatti Department of Pathology & Laboratory Medicine and Purpose of review Department of Human Genetics, David Geffen School This review is to highlight the most current mutation-targeted therapeutic approaches of Medicine at UCLA, Los Angeles, California, USA and provide insights into new developments for treating primary immunodeficiencies. Correspondence to Richard A. Gatti, MD, Department Recent findings of Pathology and Laboratory Medicine, David Geffen School of Medicine at UCLA, Los Angeles, Significant progress in mutation-targeted treatment was achieved in the past year with CA 90095-1732, USA the identification and characterization of a translational read-through compound, Tel/fax: +1 310 825 7618; e-mail: rgatti@mednet.ucla.edu PTC124. PTC124 demonstrates a new class of nontoxic bioavailable small drugs. Antisense oligonucleotide-mediated techniques such as splicing redirection, exon Current Opinion in Allergy and Clinical Immunology 2008, 8:540–546 skipping, and mismatch repair have been successfully used to correct splicing, frameshift, and missense mutations, respectively. Delivery of antisense oligonucleotides to mammalian cells, including primary leukocytes and neurons, saw great progress during the past year. Recent advances for other approaches to correct frameshift and missense mutations are also considered. Summary Primary immunodeficiencies

Journal

Current Opinion in Allergy and Clinical Immunology – Wolters Kluwer Health

Published: Dec 1, 2008

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