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Evolving adeno-associated viruses for gene transfer to the kidney via cross-species cycling of capsid libraries
Abstract The difficulty of delivering genes to the kidney has limited the translation of genetic medicines, particularly for the more...
In vivo engineering of murine T cells using the evolved adeno-associated virus variant Ark313
Summary Genetic engineering of TÂ cells in mouse models is essential for investigating immune mechanisms. We aimed to develop an approach...
Sub-genomic flaviviral RNA elements increase the stability and abundance of recombinant AAV vector transcripts
Abstract Many viruses have evolved structured RNA elements that can influence transcript abundance and translational efficiency, and help...
Engineered IgM and IgG cleaving enzymes for mitigating antibody neutralization and complement activation in AAV gene transfer
Abstract Systemic dosing of adeno-associated viral (AAV) vectors poses potential risk of adverse side effects including complement...
Repurposing CRISPR-Cas13 systems for robust mRNA trans-splicing
Type VI CRISPR enzymes have been developed as programmable RNA-guided Cas proteins for eukaryotic RNA editing. Notably, Cas13 has been...
Capsid-mediated control of adeno-associated viral transcription determines host range
Adeno-associated virus (AAV) is a member of the genus Dependoparvovirus, which infects a wide range of vertebrate species. Here, we...
An evolved AAV variant enables efficient geneticengineering of murine T cells
Precise targeting of large transgenes to T cells using homology-directed repair has been transformative for adoptive cell therapies and T...
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome...
Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to...
Epigenetic Silencing of Recombinant Adeno-associated Virus Genomes by NP220 and the HUSH Complex
The single-stranded DNA genome of adeno-associated viruses (AAV) undergoes second-strand synthesis and transcription in the host cell...
The membrane associated accessory protein is an adeno-associated viral egress factor
Adeno-associated viruses (AAV) rely on helper viruses to transition from latency to lytic infection. Some AAV serotypes are secreted in a...
Receptor Switching in Newly Evolved Adeno-associated Viruses
Adeno-associated viruses utilize different glycans and the AAV receptor (AAVR) for cellular attachment and entry. Directed evolution has...
Rescuing AAV Gene Transfer from Neutralizing Antibodies
Preexisting humoral immunity to recombinant adeno-associated virus (AAV) vectors restricts the treatable patient population and efficacy...
Humanizing AAV capsids through directed evolution
Coevolution of Adeno-associated Virus Capsid Antigenicity and Tropism through a Structure-Guided Approach Approaches to engineer...
Crossing the blood-brain barrier
Mapping structural features of certain AAVs that enable transport across the brain vasculature Effective gene delivery to the CNS by...
Using a CRISPR Screen to identify AAV Restriction Factor
A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes...
AAV Transduction in CNS influenced by Sialic Acid Dependence
Modulation of Sialic Acid Dependence Influences the Central Nervous System Transduction Profile of Adeno-associated Viruses Central...
Ring Finger Protein a Regulator of AAV Transcription
Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription Adeno-associated viruses (AAV) are...
Engineering circular RNAs
Tissue-Dependent Expression and Translation of Circular RNAs with Recombinant AAV Vectors In Vivo Circular RNAs (circRNAs) are...
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