Evidence map›Paper›PMID 28988711›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018

In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the SVZ.

David S Ojala, Sabrina Sun, Jorge L Santiago-Ortiz, Mikhail G Shapiro, Philip A Romero, David V Schaffer

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

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 63 papers.

0numbers the graph read from it
0cells of the map it votes in
63citing papers in PubMed
5.7field-weighted citation impact, top 4% 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

63 citing papers in PubMed, 112 citations in OpenAlex.

  1. Article
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  3. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
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  4. Article
  5. Scaling DNA engineering.Trends in biotechnology · 2025
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  6. Review
  7. Review
  8. AAV vector development, back to the future.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  9. Review
  10. Just a SNP away: The future ofCell insight · 2025
    Review
  11. Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Review
  17. Article
  18. Review
  19. Article
  20. Article

3 more citing papers are in PubMed but not listed here.

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.

David S OjalaDepartment of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA, USA.
Sabrina SunDepartment of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA, USA.
Jorge L Santiago-OrtizDepartment of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA, USA.
Mikhail G ShapiroDepartment of Bioengineering, University of California, Berkeley, Berkeley, CA, USA.
Philip A RomeroDepartment of Biochemistry, University of Wisconsin, Madison, WI, USA.
David V SchafferDepartment of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA, USA; Department of Bioengineering, University of California, Berkeley, Berkeley, CA, USA; The Helen Wills Neuroscience Institute, University of California, Berkeley, Berkeley, CA, USA; Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, USA. Electronic address: schaffer@berkeley.edu.
University of California, Berkeley · USUniversity of Wisconsin–Madison · US

Funding

Directed Evolution of Adeno-Associated Virus for Retinal Gene TherapyR01EY022975 · NEI · UNIVERSITY OF CALIFORNIA BERKELEY · PI FLANNERY, JOHN GERARD, SCHAFFER, DAVID V · 2014 to 2018
$2.1M
NEI NIH HHS R01 EY022975
6 · The paper itself

Abstract

Directed evolution continues to expand the capabilities of complex biomolecules for a range of applications, such as adeno-associated virus vectors for gene therapy; however, advances in library design and selection strategies are key to develop variants that overcome barriers to clinical translation. To address this need, we applied structure-guided SCHEMA recombination of the multimeric adeno-associated virus (AAV) capsid to generate a highly diversified chimeric library with minimal structural disruption. A stringent in vivo Cre-dependent selection strategy was implemented to identify variants that transduce adult neural stem cells (NSCs) in the subventricular zone. A novel variant, SCH9, infected 60% of NSCs and mediated 24-fold higher GFP expression and a 12-fold greater transduction volume than AAV9. SCH9 utilizes both galactose and heparan sulfate as cell surface receptors and exhibits increased resistance to neutralizing antibodies. These results establish the SCHEMA library as a valuable tool for directed evolution and SCH9 as an effective gene delivery vector to investigate subventricular NSCs.

Indexed as

Genetic EngineeringTransduction, GeneticAnimalsCapsid ProteinsDependovirusGalactoseGene LibraryGenetic TherapyGenetic VectorsGene Transfer TechniquesGenome, ViralHeparan SulfateHumansImaging, Three-DimensionalLateral VentriclesMiceCapsid ProteinsGalactoseHeparan Sulfateadeno-associated viruscapsid engineeringCNSdirected evolutiongene therapyneural stem cellsubventricular zone

Identifiers

PMID28988711
PMCPMC5762983
OpenAlexW2753180868

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.