September 26, 2013Human Gene Therapy Clinical DevelopmentOpen Access

Biodistribution of AAV8 Vectors Expressing Human Low-Density Lipoprotein Receptor in a Mouse Model of Homozygous Familial Hypercholesterolemia

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Population

Mouse model of homozygous familial hypercholesterolemia (hoFH)

Design

Preclinical

Follow-up

up to 180 days

Authors

SCShu‐Jen ChenNational Health Research Institutes JSJulio SanmiguelMassachusetts Eye and Ear Infirmary MLMartin LockWithernsea Hospital

Discussion

Member takes

Implication

Favorable liver biodistribution in hoFH mice supports further vector optimization; leaves open human translation and safety.

Structured PICO

Population

Mouse model of homozygous familial hypercholesterolemia (hoFH)

Intervention

Intravenous administration of AAV8.TBG.hLDLR (recombinant adeno-associated viral vector serotype 8 expressing human low-density lipoprotein receptor driven by thyroxin-binding globulin promoter)

Outcome

Vector biodistribution in organs and circulating blood over time (up to 180 days)safety

AAV8.TBG.hLDLR gene therapy demonstrates a favorable biodistribution profile with high liver tropism in a mouse model of hoFH, supporting its advancement to clinical trials.

Cite This Study

Chen et al. (2013) studied this question.

synapsesocial.com/papers/6a8bf100df3a83baf8cab1be https://doi.org/10.1089/humc.2013.082

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Biodistribution of AAV8 Vectors Expressing Human Low-Density Lipoprotein Receptor in a Mouse Model of Homozygous Familial Hypercholesterolemia

Discussion

Member takes

Implication

Structured PICO

Cite This Study

Biodistribution of AAV8 Vectors Expressing Human Low-Density Lipoprotein Receptor in a Mouse Model of Homozygous Familial Hypercholesterolemia

Discussion

Member takes

Implication

Structured PICO

Cite This Study

Also Consider

Also Consider