Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-modified AAV Vector

Kalpita R. Karan; Sławomir Andrzejewski; Katie M. Stiles; Neil R. Hackett; Ronald G. Crystal

doi:10.1089/hum.2024.112

September 25, 2024Human Gene Therapy

Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-modified AAV Vector

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Key Points

Engineered AAV.S2 capsids successfully deliver microRNAs to suppress APOE4 expression in the central nervous system, targeting a critical Alzheimer's disease risk factor.
The modified AAV.S2 vector provided 2.31 ± 0.37-fold higher expression of miRNA compared to parental AAVrh.10 two weeks after intra-hippocampus administration.
Screening peptide modifications in cell lines enabled hippocampus delivery of mir2A and mirN4 in TRE4 mice; mouse model limits require further preclinical testing.

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Implication

Preclinical study demonstrates suppression of CNS APOE4 by an engineered AAV vector in mice, highlighting a potential gene therapy approach for Alzheimer's disease.

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Cite This Study

Karan et al. (2024) studied this question.

synapsesocial.com/papers/68e5766bb6db6435875167df https://doi.org/10.1089/hum.2024.112

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Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-modified AAV Vector

Key Points

No abstract yet

Implication

Discussion

Cite This Study

Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-modified AAV Vector

Key Points

No abstract yet

Implication

Discussion

Cite This Study

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