alexa Gene Therapy to Treat Inherited Retinal Diseases | Open Access Journals
ISSN: 2161-1041
Hereditary Genetics: Current Research
Like us on:
Make the best use of Scientific Research and information from our 700+ peer reviewed, Open Access Journals that operates with the help of 50,000+ Editorial Board Members and esteemed reviewers and 1000+ Scientific associations in Medical, Clinical, Pharmaceutical, Engineering, Technology and Management Fields.
Meet Inspiring Speakers and Experts at our 3000+ Global Conferenceseries Events with over 600+ Conferences, 1200+ Symposiums and 1200+ Workshops on
Medical, Pharma, Engineering, Science, Technology and Business

Gene Therapy to Treat Inherited Retinal Diseases

Ji-jing Pang*

Department of Ophthalmology, College of Medicine, University of Florida, 1600 SW Archer Road, Gainesville, FL 32610, USA

*Corresponding Author:
Ji-jing Pang
Department of Ophthalmology
College of Medicine
University of Florida, 1600 SW Archer Road
Gainesville, FL 32610, USA
Tel: 1-352-273-9341 (office)
Fax: 1-352-392-0573
E-mail: [email protected]

Received Date: November 08, 2012; Accepted Date: December 17, 2012; Published Date: December 19, 2012

Citation: Pang JJ (2013) Gene Therapy to Treat Inherited Retinal Diseases. Genetics 2:e107. doi: 10.4172/2161-1041.1000e107

Copyright: © 2013 Pang JJ. 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.

Visit for more related articles at Hereditary Genetics: Current Research

Since 1990s, more and more mutant genes that cause human diseases have been detected. At the same time, many naturally occurring animal models of the human diseases have also been found. Those animal models have similar gene mutations and show similar phenotypes as the human diseases. Those together with the great improvement of viral vectors have led to the development of a variety of therapeutic strategies and make more and more traditionally incurable inherited diseases becoming potential candidates for gene therapy.

For example, the eye has a combination of unique features, such as transparency and a highly compartmentalized anatomy, which make it particularly suitable as a target for gene therapy. After passing through transparent structures within the eye, light then reaches and interacts with the retina directly. This feature enables the visualization and accuracy of vector delivery and the subsequent non-invasive imaging and examination in vivo. The highly compartmentalized and enclosed anatomy of the eye makes the delivery of vectors to particular subsets of ocular cell types possible with minimal risk of vector dissemination to the rest of the body. The subretinal space between the retinal pigment epithelium (RPE) and photoreceptor has a relatively high degree of immunoprivilege and is thus being considered an ideal route for the delivery of vectors. Currently, the common vehicle to deliver the therapeutic gene into target retinal cells is adeno-associated viral vector (AAV). Because of the immune privilege of subretinal space, subretinal delivery of AAV vectors can efficiently target RPE and photoreceptor cells, in which most of the mutant genes locate. In recent years, different mouse models, especially naturally occurring mouse models of retinal diseases with recessive mutations have been treated with AAV vectors encoding therapeutic genes. AAV-mediated wild type gene expressions in targeted retinal cells of those models lead to obvious retinal rescues in many fields, including electrophysiology, morphology, biochemistry and behavior, proved by the current ongoing phase I/II Leber congenital Amaurosis type 2 (LCA2 with RPE65 mutation) gene therapy clinical trial. We concluded that gene therapy will be one of the focuses for the Journal of Hereditary Genetics.

Select your language of interest to view the total content in your interested language
Post your comment

Share This Article

Relevant Topics

Recommended Conferences

Article Usage

  • Total views: 11527
  • [From(publication date):
    March-2013 - Nov 19, 2017]
  • Breakdown by view type
  • HTML page views : 7754
  • PDF downloads : 3773
 

Post your comment

captcha   Reload  Can't read the image? click here to refresh

Peer Reviewed Journals
 
Make the best use of Scientific Research and information from our 700 + peer reviewed, Open Access Journals
International Conferences 2017-18
 
Meet Inspiring Speakers and Experts at our 3000+ Global Annual Meetings

Contact Us

Agri & Aquaculture Journals

Dr. Krish

[email protected]

1-702-714-7001Extn: 9040

Biochemistry Journals

Datta A

[email protected]

1-702-714-7001Extn: 9037

Business & Management Journals

Ronald

[email protected]

1-702-714-7001Extn: 9042

Chemistry Journals

Gabriel Shaw

[email protected]

1-702-714-7001Extn: 9040

Clinical Journals

Datta A

[email protected]

1-702-714-7001Extn: 9037

Engineering Journals

James Franklin

[email protected]

1-702-714-7001Extn: 9042

Food & Nutrition Journals

Katie Wilson

[email protected]

1-702-714-7001Extn: 9042

General Science

Andrea Jason

[email protected]

1-702-714-7001Extn: 9043

Genetics & Molecular Biology Journals

Anna Melissa

[email protected]

1-702-714-7001Extn: 9006

Immunology & Microbiology Journals

David Gorantl

[email protected]

1-702-714-7001Extn: 9014

Materials Science Journals

Rachle Green

[email protected]

1-702-714-7001Extn: 9039

Nursing & Health Care Journals

Stephanie Skinner

[email protected]

1-702-714-7001Extn: 9039

Medical Journals

Nimmi Anna

[email protected]

1-702-714-7001Extn: 9038

Neuroscience & Psychology Journals

Nathan T

[email protected]

1-702-714-7001Extn: 9041

Pharmaceutical Sciences Journals

Ann Jose

[email protected]

1-702-714-7001Extn: 9007

Social & Political Science Journals

Steve Harry

[email protected]

1-702-714-7001Extn: 9042

 
© 2008- 2017 OMICS International - Open Access Publisher. Best viewed in Mozilla Firefox | Google Chrome | Above IE 7.0 version
adwords