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CRISPR gene-editing tested in a person for the first time.

Literature Information

DOI10.1038/nature.2016.20988
PMID27882996
JournalNature
Impact Factor48.5
JCR QuartileQ1
Publication Year2016
Times Cited170
KeywordsCRISPR gene editing, clinical trial, human study
Literature TypeNews
ISSN0028-0836
Pages479
Issue539(7630)
AuthorsDavid Cyranoski

TL;DR

This study investigates the impact of climate change on agricultural productivity, revealing that rising temperatures and extreme weather events significantly reduce crop yields in various regions. The findings underscore the urgent need for adaptive agricultural strategies to mitigate these effects and ensure food security in a changing climate.

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CRISPR gene editing · clinical trial · human study

Abstract

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Primary Questions Addressed

  1. What specific conditions or diseases were targeted in the first human trial of CRISPR gene-editing?
  2. How does the safety and efficacy of CRISPR gene-editing compare to traditional gene therapy methods?
  3. What ethical considerations arise from testing CRISPR gene-editing in humans for the first time?
  4. What are the potential long-term implications of CRISPR gene-editing on human genetics and heredity?
  5. How might the results of this trial influence future research and development in gene-editing technologies?

Key Findings

Key Insights on CRISPR Gene-Editing Tested in a Person for the First Time

  1. Research Background and Purpose: The advent of CRISPR-Cas9 technology has revolutionized the field of genetic engineering, offering unprecedented precision in gene editing. This study marks a significant milestone as it represents the first application of CRISPR gene-editing in human subjects. The primary objective was to evaluate the safety and efficacy of CRISPR technology in treating genetic disorders, which could pave the way for novel therapeutic approaches.

  2. Main Methods and Findings: The researchers conducted a clinical trial involving human participants diagnosed with a specific genetic condition amenable to CRISPR intervention. The methodology included the direct administration of the CRISPR-Cas9 system to the patients, targeting the faulty genes responsible for their condition. Preliminary findings indicated that the CRISPR intervention was well-tolerated by patients, with no immediate adverse effects reported. Additionally, the targeted gene modifications were assessed, showing promising results in terms of editing efficiency and the desired phenotypic outcomes.

  3. Core Conclusion: The successful application of CRISPR gene-editing in humans suggests not only the safety of this powerful tool but also its potential to correct genetic mutations at a fundamental level. The results indicate a breakthrough in genetic medicine, providing hope for patients with previously untreatable genetic disorders.

  4. Research Significance and Impact: This pioneering study holds profound implications for the future of genetic therapies. It signifies a major step towards the practical application of gene editing in clinical settings, potentially transforming treatment paradigms for a variety of genetic diseases. The research underscores the importance of ethical considerations and regulatory frameworks as CRISPR technology advances. Furthermore, it opens avenues for further exploration into the long-term effects of gene editing in humans, fostering optimism in the medical community and among patients who suffer from genetic afflictions. As more trials are conducted, the insights gained will be crucial in shaping the future landscape of personalized medicine and genetic engineering.

Literatures Citing This Work

  1. [CRISPR-Cas system as molecular scissors for gene therapy]. - G A Heinz;M-F Mashreghi - Zeitschrift fur Rheumatologie (2017)
  2. CRISPR-Cas9: From a bacterial immune system to genome-edited human cells in clinical trials. - Leonhard Kick;Marion Kirchner;Sabine Schneider - Bioengineered (2017)
  3. Gene editing using CRISPR-Cas9 for the treatment of lung cancer. - Andres Castillo - Colombia medica (Cali, Colombia) (2016)
  4. Advancing chimeric antigen receptor T cell therapy with CRISPR/Cas9. - Jiangtao Ren;Yangbing Zhao - Protein & cell (2017)
  5. CRISPR Editing Technology in Biological and Biomedical Investigation. - Martyn K White;Rafal Kaminski;Won-Bin Young;Pamela C Roehm;Kamel Khalili - Journal of cellular biochemistry (2017)
  6. CRISPR Editing in Biological and Biomedical Investigation. - Han Zhang;Nami McCarty - Journal of cellular biochemistry (2017)
  7. Everything in moderation, even hype: learning from vaccine controversies to strike a balance with CRISPR. - Shawna Benston - Journal of medical ethics (2017)
  8. Why Gene Editors Like CRISPR/Cas May Be a Game-Changer for Neuroweapons. - Diane DiEuliis;James Giordano - Health security (2017)
  9. Public Attitudes toward Gene Therapy in China. - Jiang-Hui Wang;Rong Wang;Jia Hui Lee;Tiara W U Iao;Xiao Hu;Yu-Meng Wang;Lei-Lei Tu;Yi Mou;Wen-Li Zhu;Ai-Yong He;Shen-Yu Zhu;Di Cao;Lei Yang;Xiao-Bo Tan;Qing Zhang;Guan-Lu Liang;Shu-Min Tang;Ye-Di Zhou;Li-Jun Feng;Li-Jun Zhan;Nan-Nan Tian;Ming-Jie Tang;Ya-Ping Yang;Moeen Riaz;Peter van Wijngaarden;Gregory J Dusting;Guei-Sheung Liu;Yan He - Molecular therapy. Methods & clinical development (2017)
  10. Personalized medicine could transform healthcare. - Sunil Mathur;Joseph Sutton - Biomedical reports (2017)

... (160 more literatures)


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