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Published on: September 13, 2022
Gene editing in common cardiovascular diseases
Anna-Maria Lauerer1, Xurde M Caravia2, Lars S Maier1
1Department of Internal Medicine II, University Hospital Regensburg, Regensburg, Germany.
Insights
CRISPR-Cas9 gene editing shows promise for cardiovascular diseases by correcting mutations or disrupting pathogenic pathways. Challenges include delivery and immune response, but it offers a novel therapeutic avenue.
Area of Science:
- Biotechnology
- Genetics
- Cardiology
Background:
- Cardiovascular diseases (CVDs) are a major global health burden, with current treatments having limitations like side effects and short-term efficacy.
- Novel therapeutic strategies are essential to address the high morbidity and mortality associated with CVDs.
- CRISPR-Cas9 genome editing presents a promising new approach for treating cardiovascular conditions.
Purpose of the Study:
- To review CRISPR-Cas9 gene editing approaches for cardiovascular diseases.
- To discuss the advantages and disadvantages of different gene editing strategies.
- To outline the opportunities and challenges in applying CRISPR-Cas9 for CVD treatment.
Main Methods:
- Review of current literature on CRISPR-Cas9 applications in cardiovascular research.
- Analysis of gene editing strategies for correcting hereditary mutations and disrupting pathogenic signaling cascades.
- Discussion of delivery methods and potential immune responses related to CRISPR-Cas9 components.
Main Results:
- CRISPR-Cas9 can correct rare hereditary mutations causing CVDs.
- Gene editing can disrupt common pathogenic signaling pathways, offering a more generalizable approach.
- Challenges include optimizing editing efficiency, delivery systems, and managing immune responses.
Conclusions:
- CRISPR-Cas9 gene editing holds significant potential for treating cardiovascular diseases.
- Targeting common pathogenic pathways offers broader applicability than correcting rare mutations.
- Further research is needed to overcome delivery and immunogenicity challenges for clinical translation.
Abstract:
Cardiovascular diseases are the leading cause of morbidity and mortality worldwide, highlighting the high socioeconomic impact. Current treatment strategies like compound-based drugs or surgeries are often limited. On the one hand, systemic administration of substances is frequently associated with adverse side effects; on the other hand, they typically provide only short-time effects requiring daily intake. Thus, new therapeutic approaches and concepts are urgently needed. The advent of CRISPR-Cas9 genome editing offers great promise for the correction of disease-causing hereditary mutations. As such mutations are often very rare, gene editing strategies to correct them are not broadly applicable to many patients. Notably, there is recent evidence that gene editing technology can also be deployed to disrupt common pathogenic signaling cascades in a targeted, specific, and efficient manner, which offers a more generalizable approach. However, several challenges remain to be addressed ranging from the optimization of the editing strategy itself to a suitable delivery strategy up to potential immune responses to the editing components. This review article discusses important CRISPR-Cas9-based gene editing approaches with their advantages and drawbacks and outlines opportunities in their application for treatment of cardiovascular diseases.
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