Browsing by Keyword "AAV"
Now showing items 1-7 of 7
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AAV TRANSDUCTION TRACING IN HEMATOPOIETIC CELLS BY TARGETED SCARRING OF GENOMIC SAFE HARBORS
(2023-05-10)AAV is a potent and broadly used gene delivery platform with potential applications to treat many genetic diseases, including incurable diseases of hematopoietic cells and the immune system. As this viral vector becomes ... -
AAV-associated toxicity caused by transgene expression in the retinal pigment epithelium
(2024-01-26)The field of AAV gene therapy has recently experienced multiple clinical successes, and AAV continues to be a relatively safe viral vector. Nevertheless, there is a long history of toxic side effects at higher doses, ... -
In Vivo Delivery of Precision Genome Editing Agents
(2023-03-14)The capabilities of researchers to modify the mammalian genome has expanded greatly in the last decade, but translation of these editing technologies to therapeutics is limited by the lack of efficient means to deliver the ... -
Investigating the contribution of Fc function to effective antibody-based HIV prevention
(2021-01-08)Broadly neutralizing antibodies (bNAbs) show significant potential for HIV prevention stemming from their capacity to neutralize many of the genetically diverse HIV strains currently circulating worldwide. While these ... -
Longitudinal Evolutionary Dynamics of HIV Escape from Broadly Neutralizing Antibodies
(2021-03-05)We conducted experimental in vivo evolution of HIV to understand how the virus mutates to resist broadly neutralizing antibodies (bNAbs). In the first part, we longitudinally sequenced HIV escape from bNAbs in humanized ... -
NGS-based Engineering of Potent Muscle-Tropic Gene Therapy Vectors and Synthetic Controls for Viral Surveillance
(2022-05-12)Advanced genomic approaches have impacted nearly every field of biology in the past decade, and present an especially powerful tool to detect, study, and engineer viruses. Through the application of novel technologies ... -
Novel High-Throughput Methods to Study AAV Immunity Using Combinatorial Capsid Libraries
(2021-03-05)Adeno-associated virus (AAV) is a helper-dependent, nonpathogenic virus that has shown significant promise as a gene therapy vector. As a result of abundant natural AAV infections, a majority of the population harbors ...