alexa Hutchinson Gilford Progeria Syndrome: A Therapeutic Approach via Adenoviral Delivery of CRISPR/cas Genome Editing System | OMICS International | Abstract
ISSN: 2157-7412

Journal of Genetic Syndromes & Gene Therapy
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Review Article

Hutchinson Gilford Progeria Syndrome: A Therapeutic Approach via Adenoviral Delivery of CRISPR/cas Genome Editing System

Walter Arancio*, Swonild Ilenia Genovese, Giuseppe Pizzolanti and Carla Giordano
Section of Cardio-Respiratory and Endocrine-Metabolic Diseases, Biomedical Department of Internal and Specialist Medicine (Di.Bi.M.I.S.), University of Palermo, Piazza delle Cliniche 2, 90127 Palermo, Italy
Corresponding Author : Walter Arancio
Section of Cardio-Respiratory and Endocrine-Metabolic Diseases
Biomedical Department of Internal and Specialist Medicine (Di.Bi.M.I.S.)
University of Palermo, Piazza delle Cliniche 2, 90127 Palermo, Italy
Tel: +390916552138
E-mail: [email protected]
Received October 10, 2014; Accepted December 22, 2014; Published December 29, 2014
Citation: Arancio W, Genovese SI, Pizzolanti G, Giordano C (2015) Hutchinson Gilford Progeria Syndrome: A Therapeutic Approach via Adenoviral Delivery of CRISPR/cas Genome Editing System. J Genet Syndr Gene Ther 6:256. doi:10.4172/2157-7412.1000256
Copyright: © 2014 Arancio W, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Abstract

Hutchinson-Gilford Progeria Syndrome (HGPS) is a rare human genetic disease caused by mutations in the LMNA gene. LMNA codes for structural components of the nuclear lamina. Alterations of nuclear lamina lead to a very variable class of diseases known as laminopathies. In detail, HGPS manifests a severe premature ageing phenotype due to the accumulation of a dominant negative form of lamin-A called progerin. With current treatments, the life expectancy of HGPS patients does not exceed their second decade. Death is usually due to cardiovascular complications. Recently, a new technology for mammals in vivo gene editing has been developed: the clustered regularly interspaced short palindromic repeats/Cas protein (CRISPR/Cas) system. The CRISPR/Cas technology permits to edit the genome at specific loci. Even if the CRSIPR/Cas constructs are transiently administered to the target cells, the genome editing is permanent. The advantages of the combination of non-integrating transient vectors in combination with the CRISPR/Cas constructs could give rise to a secure approach for the treatment of disease of genetic origin, especially those caused by dominant negative mutations, such as HGPS. A potential application of non-integrating transient vectors carrying CRISPR/Cas constructs for the treatment of HGPS will be discussed in detail.

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