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Gene Therapy- Challenges & Success

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Gene Therapy- Challenges & Success

Ms. Snehal D. Jadhav | Mr. Jeevan R. Rajguru | Ms. Hina U. Momin | Dr. Mrunal K. Shirsat

Ms. Snehal D. Jadhav | Mr. Jeevan R. Rajguru | Ms. Hina U. Momin | Dr. Mrunal K. Shirsat "Gene Therapy- Challenges & Success" Published in International Journal of Trend in Scientific Research and Development (ijtsrd), ISSN: 2456-6470, Volume-4 | Issue-3, April 2020, pp.870-874, URL: https://www.ijtsrd.com/papers/ijtsrd30612.pdf

Gene therapy is a contemporary therapeutic intervention with recent positive results and regulatory approvals either completed or expected in the next several years for various conditions. The evolving view is that gene therapy will ultimately offer hope across a range of otherwise debilitating or difficult to-treat conditions. The renaissance in gene therapy has seen major development of both non-viral and viral vectors and accelerated preclinical studies and clinical trials. It is therefore timely to address the progress in gene therapy through a special issue presenting reviews on non-viral and viral vectors including relevant updates on applications on herpes simplex virus (HSV) and adeno-associated virus (AAV) vectors. Thus, the purpose of this review is to summarize the general concepts of gene therapy with a specific focus on monogenic rare disease in hematology and central nervous system disorders where burgeoning therapies are currently entering clinical investigations and approaching regulatory approval.

Gene, viral vector, non viral vector, operon, cistron, muton, expression

Volume-4 | Issue-3, April 2020
IJTSRD | www.ijtsrd.com | E-ISSN 2456-6470
Copyright © 2019 by author(s) and International Journal of Trend in Scientific Research and Development Journal. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (CC BY 4.0) (http://creativecommons.org/licenses/by/4.0)

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