Evidence map›Paper›PMID 38977637›Full record

ArticleTranslational stroke research2025

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

Alka Yadav, Rich Liang, Kelly Press, Annika Schmidt, Zahra Shabani, Kun Leng, Calvin Wang, Abinav Sekhar, Joshua Shi, Garth W Devlin and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Translational stroke research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Article
  2. What's new in hereditary hemorrhagic telangiectasia?Hematology. American Society of Hematology. Education Program · 2025
    Review
  3. Article
  4. Review
  5. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

13 authors.

Alka YadavCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Rich LiangCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Kelly PressCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Annika SchmidtCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Zahra ShabaniCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Kun LengCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Calvin WangCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Abinav SekharCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Joshua ShiCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA.
Garth W DevlinDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
Trevor J GonzalezDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
Aravind AsokanDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
Hua SuCenter for Cerebrovascular Research, Department of Anesthesia and Perioperative Care, University of California, 2540 23Rd Street, Box 1363, San Francisco, CA, 94143, USA. hua.su@ucsf.edu.ORCID http://orcid.org/0000-0003-1566-9877

Funding

Uncover the Pathogenesis of Brain Arteriovenous MalformationR01NS027713 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 1990 to 2023
$8.6M
Reduction of Brain AVM Severity through Inhibition of Pathogenic AngiogenesisR01NS112819 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 2020 to 2024
$2.9M
Cell type-specific influences on HHT pathogenesisR01HL122774 · NHLBI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 2015 to 2018
$1.6M
NHLBI NIH HHS R01 HL122774NIH HHS (NS027713 and NS112819)NINDS NIH HHS R01 NS027713NINDS NIH HHS R01 NS112819
6 · The paper itself

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 (i.v.) delivery of soluble Feline McDonough Sarcoma (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 variants and delivery methods that can best transduce brain and nasal tissue with an optimal transduction profile, we compared 3 engineered AAV capsids (AAV.cc47, AAV.cc84, and AAV1RX) with AAV9. A single-stranded CBA promoter driven tdTomato transgene was packaged in these capsids and delivered i.v. or intranasally (i.n.) to wild-type mice. A CMV promoter driven Alk1 transgene was packaged into AAV.cc84 and delivered to PdgfbiCre;Alk1

Indexed as

CapsidDependovirusGenetic TherapyGene Transfer TechniquesTelangiectasia, Hereditary HemorrhagicActivin Receptors, Type IIAnimalsGenetic VectorsHumansMiceMice, Inbred C57BLActivin Receptors, Type IIAdeno-associated viral vectorAlk1Brain arteriovenous malformationGene therapyHereditary hemorrhagic telangiectasia

Identifiers

PMID38977637
PMCPMC11968179

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.