人类基因治疗中的监管问题。

Regulatory concerns in human gene therapy.

作者信息

Epstein S L

机构信息

Molecular Immunology Laboratory, Division of Biochemistry and Biophysics, FDA, CBER, Bethesda, MD 20892.

出版信息

Hum Gene Ther. 1991 Fall;2(3):243-9. doi: 10.1089/hum.1991.2.3-243.

DOI:10.1089/hum.1991.2.3-243

PMID:1751592

Abstract

Gene therapy in humans is now being undertaken in an investigational setting. Such therapy involves the administration of biological products to human patients. A document entitled, "Points to Consider in Human Somatic Cell Therapy and Gene Therapy" has been prepared by the Center for Biologics Evaluation and Research (CBER) of the Food and Drug Administration (FDA) and is published elsewhere in this issue. This paper provides explanatory material about the CBER regulatory process and the scientific and regulatory basis for the requests for data in that document.

摘要

目前，人类基因治疗正处于研究阶段。这种治疗涉及向人类患者施用生物制品。食品药品监督管理局（FDA）生物制品评估和研究中心（CBER）编写了一份题为《人类体细胞治疗和基因治疗需考虑的要点》的文件，该文件刊载于本期其他地方。本文提供了关于CBER监管程序以及该文件中数据要求的科学和监管依据的解释性材料。