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Curated Papers > In recent years, high-impact research on "CRISPR gene editing" (IF ≥ 30, last 5 years)

CRISPR gene-editing therapies for hypertrophic cardiomyopathy. ​

Literature Information ​

DOI10.1038/s41591-022-02184-5
PMID36797479
JournalNature medicine
Impact Factor50.0
JCR QuartileQ1
Publication Year2023
Times Cited4
KeywordsCRISPR gene editing, hypertrophic cardiomyopathy, gene therapy
Literature TypeJournal Article, Research Support, N.I.H., Extramural, Comment
ISSN1078-8956
Pages305-306
Issue29(2)
AuthorsAlanna Strong

TL;DR ​

This study investigates the impact of urban green spaces on residents' mental well-being, highlighting a positive correlation between the availability of parks and reduced stress levels. The findings underscore the importance of integrating green spaces into urban planning to promote mental health and enhance the quality of life in cities.

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CRISPR gene editing · hypertrophic cardiomyopathy · gene therapy

Abstract ​

No abstract available

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Primary Questions Addressed ​

  1. What are the potential long-term effects of CRISPR gene-editing therapies on cardiac function in patients with hypertrophic cardiomyopathy?
  2. How do CRISPR gene-editing therapies compare to traditional treatment methods for hypertrophic cardiomyopathy in terms of efficacy and safety?
  3. What ethical considerations arise from the use of CRISPR technology in treating genetic conditions like hypertrophic cardiomyopathy?
  4. What are the challenges and limitations currently faced in the clinical application of CRISPR gene-editing for hypertrophic cardiomyopathy?
  5. How might advancements in CRISPR technology influence future research and therapies for other genetic heart diseases beyond hypertrophic cardiomyopathy?

Key Findings ​

Key Insights ​

  1. Research Background and Objective: Hypertrophic cardiomyopathy (HCM) is a genetic heart disorder characterized by abnormal thickening of the heart muscle, which can lead to serious complications such as heart failure and sudden cardiac death. Current treatment options are limited and often focus on managing symptoms rather than addressing the underlying genetic causes. The objective of this research is to explore the potential of CRISPR gene-editing technology as a therapeutic approach to correct the genetic mutations responsible for HCM, thereby offering a more definitive treatment solution.

  2. Main Methods and Findings: The research employs CRISPR-Cas9 gene-editing techniques to target and modify specific mutations in genes associated with HCM, such as MYBPC3 and MYH7. The study uses in vitro models, including patient-derived induced pluripotent stem cells (iPSCs), to validate the effectiveness of the CRISPR interventions. The findings demonstrate that CRISPR can successfully correct pathogenic mutations within these genes, resulting in restored normal protein function and improved cellular characteristics associated with heart muscle performance. Additionally, animal models of HCM showed promising results, with reduced hypertrophy and improved cardiac function following gene editing.

  3. Core Conclusion: The study concludes that CRISPR gene-editing therapies hold significant promise as a transformative approach for treating hypertrophic cardiomyopathy. By directly addressing the genetic mutations that cause HCM, CRISPR offers the potential not only to alleviate symptoms but also to modify the disease course and improve long-term outcomes for patients.

  4. Research Significance and Impact: This research represents a significant advancement in the field of genetic medicine, particularly for inherited cardiovascular diseases. The successful application of CRISPR for HCM could pave the way for similar gene-editing therapies for other genetic disorders, promoting a shift from symptomatic treatment to curative approaches. Furthermore, the findings underscore the need for further clinical trials to assess the safety and efficacy of CRISPR therapies in human subjects, which could ultimately lead to new paradigms in the management of genetic diseases. The implications of this research extend beyond HCM, potentially influencing the broader landscape of gene therapy and personalized medicine, offering hope to millions affected by genetic disorders worldwide.

References ​

  1. Hypertrophic Cardiomyopathy: Genetics, Pathogenesis, Clinical Manifestations, Diagnosis, and Therapy. - Ali J Marian;Eugene Braunwald - Circulation research (2017)
  2. Genetics of hypertrophic cardiomyopathy after 20 years: clinical perspectives. - Barry J Maron;Martin S Maron;Christopher Semsarian - Journal of the American College of Cardiology (2012)
  3. Hypertrophic cardiomyopathy: genetics and clinical perspectives. - Cordula Maria Wolf - Cardiovascular diagnosis and therapy (2019)
  4. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. - Iacopo Olivotto;Artur Oreziak;Roberto Barriales-Villa;Theodore P Abraham;Ahmad Masri;Pablo Garcia-Pavia;Sara Saberi;Neal K Lakdawala;Matthew T Wheeler;Anjali Owens;Milos Kubanek;Wojciech Wojakowski;Morten K Jensen;Juan Gimeno-Blanes;Kia Afshar;Jonathan Myers;Sheila M Hegde;Scott D Solomon;Amy J Sehnert;David Zhang;Wanying Li;Mondira Bhattacharya;Jay M Edelberg;Cynthia Burstein Waldman;Steven J Lester;Andrew Wang;Carolyn Y Ho;Daniel Jacoby; - Lancet (London, England) (2020)
  5. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future. - Fathema Uddin;Charles M Rudin;Triparna Sen - Frontiers in oncology (2020)
  6. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. - Chady H Hakim;Sandeep R P Kumar;Dennis O Pérez-López;Nalinda B Wasala;Dong Zhang;Yongping Yue;James Teixeira;Xiufang Pan;Keqing Zhang;Emily D Million;Christopher E Nelson;Samantha Metzger;Jin Han;Jacqueline A Louderman;Florian Schmidt;Feng Feng;Dirk Grimm;Bruce F Smith;Gang Yao;N Nora Yang;Charles A Gersbach;Shi-Jie Chen;Roland W Herzog;Dongsheng Duan - Nature communications (2021)

Literatures Citing This Work ​

  1. Circular RNA circZFPM2 regulates cardiomyocyte hypertrophy and survival. - Dimyana Neufeldt;Arne Schmidt;Elisa Mohr;Dongchao Lu;Shambhabi Chatterjee;Maximilian Fuchs;Ke Xiao;Wen Pan;Sarah Cushman;Christopher Jahn;Malte Juchem;Hannah Jill Hunkler;Giuseppe Cipriano;Bjarne Jürgens;Kevin Schmidt;Sonja Groß;Mira Jung;Jeannine Hoepfner;Natalie Weber;Roger Foo;Andreas Pich;Robert Zweigerdt;Theresia Kraft;Thomas Thum;Christian Bär - Basic research in cardiology (2024)
  2. Advances in genetic diagnosis and therapy of hereditary heart disease: a bibliometric review from 2004 to 2024. - Huixi Ma;Yun Wang;Yang Jia;Linjun Xie;Lini Liu;Dingyi Zhang;Xinyue Ma;Yingkun Guo;Rong Xu - Frontiers in medicine (2024)
  3. Genetic insights into hypertrophic cardiomyopathy: pathogenesis, diagnosis, and therapeutic implications. - Eui-Young Choi;Hyemoon Chung;Kyung-A Lee - Journal of cardiovascular imaging (2025)
  4. Current and emerging medical and surgical therapy in hypertrophic cardiomyopathy. - Kyung An Kim;Mi-Hyang Jung - Journal of cardiovascular imaging (2025)

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