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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

772
The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
772

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Harnessing lipid nanoparticles for efficient CRISPR delivery.

Jingyue Yan1, Diana D Kang1, Yizhou Dong1,2

  • 1Division of Pharmaceutics & Pharmacology, College of Pharmacy, The Ohio State University, Columbus, Ohio 43210, USA. dong.525@osu.edu.

Biomaterials Science
|June 11, 2021
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Summary
This summary is machine-generated.

CRISPR gene editing offers precise DNA modification, with recent trials showing promise in cancer therapy. Novel lipid nanomaterials are being developed to overcome challenges in delivering CRISPR technology effectively within the body for various diseases.

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Area of Science:

  • Biomedical research
  • Gene editing technology
  • Nanomaterial science

Background:

  • The CRISPR-Cas system has transformed biomedical research with its precise and adaptable genome editing capabilities.
  • Nobel Prize awarded for the discovery of CRISPR-Cas9 genetic scissors, highlighting its significance in altering DNA sequences.
  • Early clinical trials demonstrate the safety and feasibility of ex vivo CRISPR-edited T cells for cancer treatment.

Purpose of the Study:

  • To discuss recent advancements in novel lipid nanomaterials for CRISPR delivery systems.
  • To explore the potential therapeutic applications of these delivery systems in treating cancers, genetic disorders, and infectious diseases.
  • To address the challenges associated with specific and effective in vivo CRISPR delivery.

Main Methods:

  • Review of recent scientific literature on lipid nanomaterials for CRISPR delivery.
  • Analysis of extracellular and intracellular barriers affecting in vivo gene editing.
  • Case examples of potential therapeutic applications in oncology, genetic diseases, and infectious diseases.

Main Results:

  • Identification of novel lipid nanomaterials as promising vehicles for CRISPR delivery.
  • Demonstration of potential therapeutic benefits across diverse disease areas.
  • Highlighting strategies to overcome delivery barriers for in vivo applications.

Conclusions:

  • Lipid nanomaterials represent a significant advancement in overcoming in vivo CRISPR delivery challenges.
  • CRISPR technology, facilitated by these nanomaterials, holds substantial therapeutic potential for a range of diseases.
  • Further research and development are crucial for translating these findings into clinical practice.