HOME HELP FEEDBACK SUBSCRIPTIONS ARCHIVE SEARCH TABLE OF CONTENTS		QUICK SEARCH:   [advanced] Author: Keyword(s): Year:  Vol:  Page:

This Article Figures Only Full Text Full Text (PDF) Correspondence: Submit a response Alert me when this article is cited Alert me when Correspondence are posted Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Citing Articles Citing Articles via Google Scholar Google Scholar Articles by Mendell, J. R. Articles by Miller, A. Search for Related Content PubMed PubMed Citation Articles by Mendell, J. R. Articles by Miller, A. Related Collections Arteriovenous malformation Carotid artery dissection All Genetics

Gene transfer for neurologic disease

Agencies, policies, and process

From the Center for Neuromuscular Disorders (Dr. Mendell), Children’s Research Institute and Department of Neurology, Ohio State University, Columbus, OH; and Cell and Gene Therapies (Dr. Miller), The Biologics Consulting Group, Alexandria, VA.

Address correspondence and reprint requests to Dr. Jerry R. Mendell, Children’s Research Institute, Department of Neurology, The Ohio State University, WA 3024, 700 Children’s Drive, Columbus, OH 43205; e-mail: mendell.1{at}osu.edu

Advances in molecular biology have contributed to a growing interest in gene therapy as a form of management for neurologic diseases. However, implementation requires knowledge of the regulatory policies governing this field of research, especially in view of the greater stringency imposed by the serious adverse events affecting some patients participating in gene therapy protocols. Educational resources for neurologists, or any clinicians, who hope to serve as potential principal investigators for a gene therapy protocol are not available through any single source, requiring considerable effort to discover appropriate guidance. Summarized here are the regulatory agencies and their requirements, the phases of clinical development with emphasis on a Phase I study, and specific steps leading to an Investigational New Drug application for a biologic product to be used in a gene therapy clinical trial. The links provided to all appropriate Web sites will facilitate the process for the clinician investigator.

Received February 24, 2004. Accepted in final form July 12, 2004.