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Ocular delivery of CRISPR/Cas genome editing components for treatment of eye diseases

Wenhan Yu1, Zhijian Wu1

  • 1Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, 6 Center Drive, Room 306, Bethesda, MD 20892, USA.

Advanced Drug Delivery Reviews
|July 1, 2020
PubMed

Insights

CRISPR gene editing offers promising treatments for inherited eye diseases. This review explores ocular delivery methods, vectors, and challenges for CRISPR-Cas systems, including newer base and prime editing tools.

Area of Science:

  • Ophthalmology
  • Genetics
  • Biotechnology

Background:

  • Inherited and multifactorial ocular diseases necessitate innovative therapeutic strategies.
  • Genome editing technologies like CRISPR present significant potential for treating these conditions.

Purpose of the Study:

  • To review ocular administration routes for CRISPR/Cas components.
  • To discuss the pros and cons of viral and non-viral vectors in ocular gene delivery.
  • To summarize current research on CRISPR/Cas for ocular diseases and identify challenges.

Main Methods:

  • Literature review of ocular administration routes for CRISPR/Cas.
  • Analysis of viral (e.g., AAV) and non-viral vectors for ocular gene delivery.
  • Examination of existing studies on CRISPR/Cas applications in ophthalmology.

Main Results:

  • Adeno-associated viral (AAV) vectors are common but may elicit immune responses and off-target effects.
  • Various administration routes and vector types have advantages and disadvantages for ocular delivery.
  • CRISPR/Cas delivery faces challenges including safety, efficacy, and specificity in ocular tissues.

Conclusions:

  • Optimizing delivery vectors and routes is crucial for safe and effective CRISPR-based ocular therapies.
  • Emerging CRISPR tools like base and prime editing may offer future therapeutic avenues for eye diseases.
  • Further research is needed to overcome current challenges in applying genome editing to ocular disorders.
Keywords:
AAV vectorCRISPR/CasGene deliveryGene therapyGenome editingRetinal degeneration

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