2024
DOI: 10.21203/rs.3.rs-4469011/v1
|View full text |Cite
Preprint
|
Sign up to set email alerts
|

Evaluation of Aav Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy

Alka Yadav,
Rich Liang,
Kelly Press
et al.

Abstract: Nosebleeds and intracranial hemorrhage from brain arteriovenous malformations (bAVMs) are among the most devastating symptoms of patients with hereditary hemorrhagic telangiectasis (HHT). All available managements have limitations. We showed that intravenous delivery of soluble FMS-related tyrosine kinase 1 using an adeno-associated viral vector (AAV9-sFLT1) reduced bAVM severity of endoglin deficient mice. However, minor liver inflammation and growth arrest in young mice were observed. To identify AAV variant… Show more

Help me understand this report
View published versions

Search citation statements

Order By: Relevance

Paper Sections

Select...

Citation Types

0
0
0

Publication Types

Select...

Relationship

0
0

Authors

Journals

citations
Cited by 0 publications
references
References 43 publications
0
0
0
Order By: Relevance

No citations

Set email alert for when this publication receives citations?