Gene therapy: advances, challenges and perspectives

Author:

Gonçalves Giulliana Augusta Rangel1,Paiva Raquel de Melo Alves1

Affiliation:

1. Hospital Israelita Albert Einstein, Brazil

Abstract

ABSTRACT The ability to make site-specific modifications to the human genome has been an objective in medicine since the recognition of the gene as the basic unit of heredity. Thus, gene therapy is understood as the ability of genetic improvement through the correction of altered (mutated) genes or site-specific modifications that target therapeutic treatment. This therapy became possible through the advances of genetics and bioengineering that enabled manipulating vectors for delivery of extrachromosomal material to target cells. One of the main focuses of this technique is the optimization of delivery vehicles (vectors) that are mostly plasmids, nanostructured or viruses. The viruses are more often investigated due to their excellence of invading cells and inserting their genetic material. However, there is great concern regarding exacerbated immune responses and genome manipulation, especially in germ line cells. In vivo studies in in somatic cell showed satisfactory results with approved protocols in clinical trials. These trials have been conducted in the United States, Europe, Australia and China. Recent biotechnological advances, such as induced pluripotent stem cells in patients with liver diseases, chimeric antigen receptor T-cell immunotherapy, and genomic editing by CRISPR/Cas9, are addressed in this review.

Publisher

FapUNIFESP (SciELO)

Subject

General Medicine

Reference30 articles.

1. The gene: an intimate history;Mukherjee S,2016

2. A brief history of gene therapy;Friedmann T;Nat Genet,1992

3. Human gene therapy: a brief overview of the genetic revolution;Misra S;J Assoc Physicians India,2013

4. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV;Tebas P;N Engl J Med,2014

5. Gene therapy: what it is, what it is not and what it will be;Linden R;Estud Av,2010

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