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(Circulation Research. 2007;100:1667.)
© 2007 American Heart Association, Inc.

Editorials

National Heart, Lung, and Blood Institute Resources and Programs for Gene Therapy in Heart, Lung, and Blood Diseases

Sonia I. Skarlatos

From the Division of Cardiovascular Diseases, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Md.

Correspondence to Sonia I. Skarlatos, PhD, FAHA, Acting Director, Division of Cardiovascular Diseases, NHLBI Gene Therapy Coordinator, National Heart, Lung and Blood Institute. E-mail skarlats@nhlbi.nih.gov

Key Words: gene therapy • translational studies • clinical research • vector systems • gene transfer • NHLBI

An extract of the first 250 words of the full text is provided, because this article has no abstract.

	Introduction

 
The advent of gene therapy holds great promise but the field is still in its infancy. Advances have been made but the translation of gene therapy from the bench to the clinical application has been slow. Relatively few clinical trials of gene therapy have been initiated for heart, lung, and blood disorders, and very few are currently ongoing.

The future of gene therapy depends on our ability to translate basic research studies into clinical applications. These "translational studies" are expensive and complex, but necessary if we are to develop gene-based treatments that treat or prevent cardiovascular, lung and blood diseases. This article describes the National Heart, Lung, and Blood Institute’s (NHLBI) current gene therapy programs which have been created to help support academic research efforts to successfully translate gene therapy into viable clinical applications.

	Challenges and Barriers for Gene Therapy

 
NHLBI convened an expert Working Group in June 2005, to better understand the issues that impede progress in gene therapy research. The group was asked for their expert advice as to the status of gene therapy research and to identify critical resources needed by academic investigators to speed clinical translation of gene therapies for heart, lung, and blood diseases.¹

The Working Group Identified Three Major Research Challenges
Producing Good Manufacturing Process Vectors Reliably and Efficiently
The ability to undertake gene therapy clinical trials will require large scale production of safe and well-characterized vectors. The choices for viral vectors for many gene therapy applications are adenovirus, adeno-associated virus (AAV), retrovirus and lentivirus. Each requires dedicated facilities and technical expertise. To date, the availability of designated facilities to produce good manufacturing process (GMP)-grade vectors . . . [Full Text of this Article]