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The promise of gene therapy

Pergament, Eugene

Current Opinion in Obstetrics and Gynecology: April 2016 - Volume 28 - Issue 2 - p 132–135
doi: 10.1097/GCO.0000000000000255
PRENATAL DIAGNOSIS: Edited by Jane Chueh

Purpose of review The promise of gene therapy performed in the preimplantation and prenatal periods of pregnancy is rapidly becoming a reality. New technologies capable of making designed changes in single nucleotides make germline gene therapy possible. The article reviews the ethical and technical challenges of germline gene therapy.

Recent findings Clustered regularly interspaced short palindromic repeats and related technologies are capable of deleting and inserting specific DNA sequences in mutated genes so as to correct the targeted DNA. The ability to target specific gene mutations will offer unique opportunities to at risk families, particularly those whose genotypes prevent any chance of a normal pregnancy outcome. Other applications of gene-modifying technologies on gametes, zygotes, and embryos are likely in the near future.

Summary There will be renewed debates on the potentially controversial applications of these technologies because of their capability to genetically alter the human germline and thereby future generations

aNorthwestern Reproductive Genetics, Inc.

bDepartment of Obstetrics and Gynecology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA

Correspondence to Eugene Pergament, MD, PhD, FACMG, Northwestern Reproductive Genetics, Inc., Chicago, IL, USA. Tel: +1 312 888 9910; fax: +1 312 888 9092; e-mail:

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