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Special Issue

Treatment of Genetic Disease

Submission Deadline: June 30, 2018 (Open)                Submit Now

Guest Editor

Khue Vu Nguyen, PhD, DSc
Full Project Scientist, Departments of Medicine and Pediatrics, University of California of San Diego, San Diego, USA
E-Mail: [email protected]
Research Interests: molecular biology; genetic diseases; enzymology; biosensors; biopolymers; neurodevelopmental and neurodegenerative disorders; cancer

About This Topic

It is with great pleasure to introduce a special issue, namely “Treatment of Genetic Disease”, which is scheduled to appear this year in OBM Genetics. I cordially invite authors to contribute their excellent works to this exciting forum. Submissions are now open and will be fully considered for publication.

Genes are the building blocks of heredity. They are passed from parent to child. A genetic disease occurs when a person has one or more abnormal genes, missing genes, extra genes, inactivated genes, or overly active genes that lead to a medical condition. With the increasing ability to control infectious and nutritional diseases in developed countries, there has come the realization that genetic diseases are a major cause of disability, death, and human tragedy. Recent findings, however, show a role of epigenetic modifications, gene-gene and/or gene-environment interactions in the disease etiology. As such, in this special issue of OBM Genetics, I have selected topics that highlight the current progress in diagnostics and treatment of genetic diseases including gene, stem cell, epigenetic therapies, and discussed about future directions in the field aimed toward innovative therapies using RNA-based therapeutic or RNA as therapeutic target. To this end, specific mRNA in cells and tissues appears as an ideal target for therapeutic intervention and antisense drugs are potential treatment.



Therapies for Childhood Polycystic Kidney Disease

Ameya Patil, William E Sweeney, Ellis D. Avner
Received: October 7, 2018; Published: December 24, 2018; doi:10.21926/obm.genet.1804056


Prospects for Cure in Wilson Disease

Michelle A. Camarata, Uyen To, Michael L. Schilsky
Received: July 18, 2018; Published: August 23, 2018; doi:10.21926/obm.genet.1803030


Experimental DNA- or RNA-Directed Therapies for Trinucleotide Repeat Disease

Inês Lopes Cardoso
Received: April 17, 2018; Published: July 27, 2018; doi:10.21926/obm.genet.1803025


Special Issue: Treatment of Genetic Disease

Khue Vu Nguyen
Received: January 19, 2018; Published: February 1, 2018; doi:10.21926/obm.genet.1801012