DGK and DZHK position paper on genome editing: basic science applications and future perspective.

Area of Science:

• Molecular Biology
• Genetics
• Biotechnology

Background:

• Gene editing was historically limited but is now widely accessible due to TALENs and CRISPR/Cas.
• CRISPR/Cas systems enable advanced gain- and loss-of-function models for unbiased screening and rapid transgenic animal development.

Purpose of the Study:

• To review recent advancements in gene editing technology.
• To focus on the translational aspects and combined cellular/animal applications of gene editing.
• To provide direction for future development and address challenges in gene editing applications.

Main Methods:

• Review of expert opinions from joint conferences of the German Cardiac Society (DGK) and the German Center for Cardiovascular Research (DZHK).
• Analysis of recent progress in gene editing, including modified CRISPR/Cas systems for epigenetic interrogation and DNA complex visualization.
• Focus on translational research combining cellular and animal models.

Main Results:

• Gene editing, particularly CRISPR/Cas, has become a powerful tool for basic research, enabling deep, unbiased screens and rapid creation of transgenic models.
• Modified CRISPR/Cas systems offer precise tools for studying epigenetic regulation and visualizing DNA structures.
• Gene editing is progressing towards clinical applications, with initial trials underway.

Conclusions:

• Gene editing technologies have transitioned from a niche concept to a broadly applicable laboratory technique.
• The field is rapidly advancing, with significant potential for both basic research and clinical translation.
• Further development is needed to optimize applications and overcome existing challenges in gene editing.