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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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...
CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
CRISPR and crRNAs02:53

CRISPR and crRNAs

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.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...

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Related Experiment Video

Updated: May 12, 2026

Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection
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Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection

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Rational Design and Optimisation of CRISPR-Cas9 Delivery Systems for Targeted Genomic Transformation.

Kalpana Pandya1,2, Likshit S Jaisinghani1, Avanish Tripathi3

  • 1Department of Pharmaceutical Chemistry, School of Pharmacy & Technology Management, SVKM's NMIMS University, Mukesh Patel Technology Park, Mumbai, Maharashtra, India.

The Journal of Gene Medicine
|May 10, 2026
PubMed
Summary

CRISPR-Cas9 gene editing shows promise for Alzheimer's disease, but delivering tools across the blood-brain barrier is key. Innovations in viral, nonviral, and physical delivery methods are advancing therapies for neurological disorders.

Keywords:
CRISPR‐Cas9blood–brain barrierdelivery vectorsgene editingneurological disorders

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Last Updated: May 12, 2026

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Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Alzheimer's disease (AD) is a neurodegenerative disorder characterized by amyloid plaques and neurofibril tangles.
  • CRISPR-Cas9 gene editing offers a potential therapeutic approach for genetic disorders like AD.
  • The blood-brain barrier (BBB) presents a major obstacle for delivering gene-editing components to the brain.

Purpose of the Study:

  • To review and compare various CRISPR-Cas9 delivery vectors for neurological diseases, particularly AD.
  • To assess the efficacy of viral, nonviral, and physical delivery methods in overcoming the BBB.
  • To highlight advancements in CNS-targeted delivery strategies.

Main Methods:

  • Comparison of viral vectors (AAVs, LVs), nonviral vectors (nanoparticles, LNPs), and physical methods (electroporation, microinjection).
  • Evaluation of vector properties including transduction efficiency, BBB permeability, immunogenicity, and safety profiles.
  • Analysis of novel strategies for enhanced brain penetration, such as receptor-mediated transcytosis.

Main Results:

  • Viral vectors like AAVs and LVs show high efficiency and BBB permeability, with AAVs favored for low immunogenicity and neuronal tropism.
  • Nonviral vectors are less immunogenic but require optimization for BBB crossing and endosomal escape.
  • Targeted delivery strategies and engineered vectors demonstrate improved brain penetration and therapeutic benefits in AD models.

Conclusions:

  • CRISPR-Cas9 delivery to the brain faces challenges, but ongoing vector innovation is crucial for clinical translation.
  • Effective delivery systems are essential for realizing the therapeutic potential of gene editing in Alzheimer's disease and other neurological disorders.
  • Addressing off-target effects, immune responses, and regulatory issues is vital for advancing CRISPR-Cas9 therapies.