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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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A multifunctional AAV-CRISPR-Cas9 and its host response.

Wei Leong Chew1,2, Mohammadsharif Tabebordbar2,3, Jason K W Cheng3

  • 1Department of Genetics, Harvard Medical School, Boston, Massachusetts, USA.

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Summary

Adeno-associated virus (AAV)-split-Cas9 enables genome engineering in mice, overcoming AAV delivery challenges for gene therapy. This platform shows promise for safe and effective therapeutic applications.

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Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Biotechnology

Background:

  • CRISPR-Cas9 gene editing via adeno-associated virus (AAV) is promising for therapy but limited by immunogenicity and payload size.
  • Developing safe and efficient AAV delivery systems is crucial for advancing genome therapeutics.

Purpose of the Study:

  • To demonstrate genome engineering in postnatal mice using a novel AAV-split-Cas9 platform.
  • To evaluate the platform's efficacy, safety, and clinical translatability by analyzing key parameters.

Main Methods:

  • Utilized AAV-split-Cas9 for genome editing, transcriptional regulation, and other applications in postnatal mice.
  • Assessed viral biodistribution, organ-specific editing efficiencies, antigenicity, and immunological responses.
  • Monitored physiological outcomes and cellular damage in vivo.

Main Results:

  • The AAV-split-Cas9 platform facilitated genome engineering with customizable applications.
  • Host responses were observed with distinct cellular and molecular signatures.
  • Crucially, the system did not induce significant in vivo cellular damage, unlike alternative methods.

Conclusions:

  • AAV-split-Cas9 offers a versatile platform for genome therapeutics, addressing limitations of conventional AAV-CRISPR-Cas9 delivery.
  • The study provides critical insights into parameters affecting efficacy and safety for clinical translation.
  • This work lays the groundwork for developing advanced genome engineering therapies.