Evidence map›Paper›PMID 42137583›Full record

ArticleMolecular therapy. Advances2026

Consequences of human genetic variations in

Jenifer M Vasquez, Hailey L Mackey, Mahboubeh R Rostami, Philip L Leopold, Ronald G Crystal

Abstract read
In one paragraph

Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Jenifer M VasquezDepartment of Genetic Medicine, Weill Cornell Medicine, New York, NY, USA.
Hailey L MackeyDepartment of Genetic Medicine, Weill Cornell Medicine, New York, NY, USA.
Mahboubeh R RostamiDepartment of Genetic Medicine, Weill Cornell Medicine, New York, NY, USA.
Philip L LeopoldDepartment of Genetic Medicine, Weill Cornell Medicine, New York, NY, USA.
Ronald G CrystalDepartment of Genetic Medicine, Weill Cornell Medicine, New York, NY, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno-associated virus (AAV) vectors mediate persistent gene expression in non-proliferating cells, but patients receiving identical vector doses exhibit variable responses. Our prior report of 69,442 genetic variants among 62 genes encoding host proteins affecting AAV gene transfer suggested that naturally occurring human genetic variants might modify AAV efficacy. We assessed 15 predicted deleterious variants of

Indexed as

AAV infection pathwayAAVRAAV serotypesadeno-associated virus receptorgene therapygene transfergenomic variantsKIAA0319Lpharmacogenomics

Identifiers

PMID42137583
PMCPMC13148924

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.