Article

Two engineered AAV capsid variants for efficient transduction of human cortical neurons directly converted from iPSC

2021-09-06

Abstract excerpt

Recombinant adeno-associated virus (AAV) is the most widely used vector for gene therapy in clinical trials. To increase transduction efficiency and specificity, novel engineered AAV variants with modified capsid sequences are evaluated in human cell cultures and non-human primates. In the present study, we tested two novel AAV capsid variants, AAV2-NNPTPSR and AAV9-NVVRSSS, in human cortical neurons, which were d...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: c561f8d8-90cc-5e10-95e1-aa56019469e5
DOI: 10.1101/2021.09.06.459070

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Two engineered AAV capsid variants for efficient transduction of human cortical neurons directly converted from iPSCDOI 10.1101/2021.09.06.459070Read

Comprehensive investigation of AAV tropism across human iPSC-derived neuronal subtypesSemantic proximity Read

Identification of Adeno-Associated Virus Variants for Gene Transfer Into Human Neural Cell Types by Parallel Capsid ScreeningSemantic proximity Read

Common AAV gene therapy vectors show indiscriminate transduction of living human brain cell typesSemantic proximity Read

Cell specificity of adeno-associated virus (AAV) serotypes in human cortical organoidsSemantic proximity Read

Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates, and humansSemantic proximity Read

Two miRNAs Efficiently Reprogram Human Fibroblasts into SST InterneuronsSemantic proximity Read

Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS VectorSemantic proximity Read

Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice.Semantic proximity Read

Alphaviruses: Semliki Forest Virus and Sindbis Virus Vectors for Gene Transfer into NeuronsSemantic proximity Read

Poor sensitivity of iPSC-derived neural progenitors and glutamatergic neurons to SARS-CoV-2Semantic proximity Read

Adeno-associated virus 9 (AAV9) viral proteins VP1, VP2, and membrane-associated accessory protein (MAAP) differentially influence in vivo transgene expressionSemantic proximity Read

Efficient Gene Expression in Human Stem Cell Derived-Cortical Organoids Using Adeno Associated VirusSemantic proximity Read