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Journal article
What's in a cure: designing a broad-spectrum HIV gene therapy
Current opinion in HIV & AIDS, v 19(3), pp 150-156
04 Mar 2024
PMID: 38547339
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
The leading gene editing strategy for a human immunodeficiency virus type 1 (HIV-1) cure involves the delivery of SaCas9 and two guide RNAs (gRNAs) in an adeno-associated viral (AAV) vector. As a dual-component system, CRISPR is targeted to a genetic locus through the choice of a Cas effector and gRNA protospacer design pair. As CRISPR research has expanded in recent years, these components have been investigated for utilization in cure strategies, which will be discussed in this article.
Type II SpCas9 and SaCas9 have been the leading Cas effectors across gene editing therapeutics to date. Additionally, extensive research has expanded the potential to multiplex gRNAs and target them effectively to the highly genetically diverse HIV-1 provirus. More recently, the Type V family of Cas12 effectors opens a new opportunity to use a smaller Cas protein for packaging into an AAV vector with multiplexed gRNAs.
In understanding the individual components of a CRISPR/Cas therapeutic cure for HIV-1, it is important to know that the currently used strategies can be improved upon. Future areas will include alternative smaller Cas effectors, multiplexed gRNAs designs, and/or alternative delivery modalities.
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Details
- Title
- What's in a cure: designing a broad-spectrum HIV gene therapy
- Creators
- Rachel E Berman - Drexel UniversityWill Dampier - Drexel UniversityMichael R Nonnemacher - Drexel UniversityBrian Wigdahl - Drexel University
- Publication Details
- Current opinion in HIV & AIDS, v 19(3), pp 150-156
- Publisher
- Lippincott
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Microbiology and Immunology
- Web of Science ID
- WOS:001233576900008
- Scopus ID
- 2-s2.0-85189861926
- Other Identifier
- 991021864438404721
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- Collaboration types
- Domestic collaboration
- Web of Science research areas
- Immunology
- Infectious Diseases