Evidence map›Paper›PMID 38115579›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2024

Evolving membrane-associated accessory protein variants for improved adeno-associated virus production.

Adam J Schieferecke, Hyuncheol Lee, Aleysha Chen, Vindhya Kilaru, Justin Krish Williams, David V Schaffer

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
3.1field-weighted citation impact, top 8% of its field
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

10 citing papers in PubMed, 10 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Review
  5. Advances in AAV capsid engineering: Integrating rational design, directed evolution and machine learning.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  6. Article
  7. Article
  8. Article
  9. Review
  10. 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

6 authors at 2 institutions in 1 country.

Adam J SchiefereckeDepartment of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; California Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, CA 94720, USA.
Hyuncheol LeeCalifornia Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, CA 94720, USA.
Aleysha ChenDepartment of Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA.
Vindhya KilaruDepartment of Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA.
Justin Krish WilliamsDepartment of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA.
David V SchafferDepartment of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA; California Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, CA 94720, USA; Department of Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA; Department of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA 94720, USA; Helen Wills Neuroscience Institute, University of California, Berkeley, Berkeley, CA 94720, USA. Electronic address: schaffer@berkeley.edu.
University of California, Berkeley · USQB3 · US

Funding

Directed Evolution of Novel AAVs and Regulatory Elements for Selective Microglial Gene ExpressionR01NS126397 · NINDS · UNIVERSITY OF CALIFORNIA BERKELEY · PI Tomasz Nowakowski, DAVID V SCHAFFER · 2023 to 2026
$3.2M
Machine Learning Augmented Discovery of AAV Capsids for Cell Type Specific Access into Human Neurons and GliaUF1MH130700 · NIMH · UNIVERSITY OF CALIFORNIA BERKELEY · PI NOWAKOWSKI, TOMASZ, SCHAFFER, DAVID V · 2022 to 2022
$2.9M
NIMH NIH HHS UF1 MH130700NINDS NIH HHS R01 NS126397
6 · The paper itself

Abstract

Manufacturing sufficient adeno-associated virus (AAV) to meet current and projected clinical needs is a significant hurdle to the growing gene therapy industry. The recently discovered membrane-associated accessory protein (MAAP) is encoded by an alternative open reading frame in the AAV cap gene that is found in all presently reported natural serotypes. Recent evidence has emerged supporting a functional role of MAAP in AAV egress, although the underlying mechanisms of MAAP function remain unknown. Here, we show that inactivation of MAAP from AAV2 by a single point mutation that is silent in the VP1 open reading frame (ORF) (AAV2-ΔMAAP) decreased exosome-associated and secreted vector genome production. We hypothesized that novel MAAP variants could be evolved to increase AAV production and thus subjected a library encoding over 1 × 10

Indexed as

Capsid ProteinsDependovirusCapsidGenetic VectorsSerogroupTransgenesCapsid ProteinsAAV2AAV6AAV9adeno-associated virusdirected evolutiongene therapyMAAPmanufacturingmembrane-associated accessory protein

Identifiers

PMID38115579
PMCPMC10861973
OpenAlexW4389903977

What OpenQuestion holds

Textmetadata
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.