将治疗性基因组编辑推向全球临床试验和医学。

IF 3.7 4区生物学 Q2 GENETICS & HEREDITY

CRISPR Journal Pub Date : 2025-05-21 DOI:10.1089/crispr.2025.0049

Kiran Musunuru, Fyodor Urnov

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引用次数: 0

摘要

将基于crispr的疗法从发现到临床试验给病人用药，再到最终获得批准，需要经历一个充满挑战的高潮和低谷。在这次采访中，内科科学家Kiran Musunuru和基因组编辑Fyodor Urnov回顾了过去20年来他们在该领域的非临床和临床项目，当前的创新前景，以及他们对未来前景的看法。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine.

Moving CRISPR-based therapies from discovery to dosing patients in clinical trials and ultimately to approval involves navigating a challenging terrain of highs and lows. In this interview, physician-scientist Kiran Musunuru and genome editor Fyodor Urnov reflect on the past 20 years of their nonclinical and clinical programs in the field, the current landscape of innovation, and what they see on the horizon.

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来源期刊

CRISPR Journal Biochemistry, Genetics and Molecular Biology-Biotechnology

CiteScore

6.30

自引率

2.70%

发文量

期刊介绍： In recognition of this extraordinary scientific and technological era, Mary Ann Liebert, Inc., publishers recently announced the creation of The CRISPR Journal -- an international, multidisciplinary peer-reviewed journal publishing outstanding research on the myriad applications and underlying technology of CRISPR. Debuting in 2018, The CRISPR Journal will be published online and in print with flexible open access options, providing a high-profile venue for groundbreaking research, as well as lively and provocative commentary, analysis, and debate. The CRISPR Journal adds an exciting and dynamic component to the Mary Ann Liebert, Inc. portfolio, which includes GEN (Genetic Engineering & Biotechnology News) and more than 80 leading peer-reviewed journals.