Evidence map›Paper›PMID 22888960›Full record

ArticleHuman gene therapy2012

Capsid-modified adenoviral vectors for improved muscle-directed gene therapy.

Kilian Guse, Masataka Suzuki, Gautam Sule, Terry K Bertin, Henna Tyynismaa, Sofia Ahola-Erkkilä, Donna Palmer, Anu Suomalainen, Philip Ng, Vincenzo Cerullo and 2 more

Abstract read
In one paragraph

Article in Human gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
2.3field-weighted citation impact, top 11% 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

18 citing papers in PubMed, 27 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Article
  6. Pluripotent stem cell therapy for retinal diseases.Annals of translational medicine · 2021
    Review
  7. Article
  8. Review
  9. High-Capacity Adenoviral Vectors: Expanding the Scope of Gene Therapy.International journal of molecular sciences · 2020
    Review
  10. Article
  11. Article
  12. Article
  13. Development of Novel Adenoviral Vectors to Overcome Challenges Observed With HAdV-5-based Constructs.Molecular therapy : the journal of the American Society of Gene Therapy · 2016
    Review
  14. Article
  15. Review
  16. Article
  17. Review
  18. 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

12 authors at 3 institutions in 2 countries.

Kilian GuseDepartment of Human and Molecular Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Masataka Suzuki
Gautam Sule
Terry K Bertin
Henna Tyynismaa
Sofia Ahola-Erkkilä
Donna Palmer
Anu Suomalainen
Philip Ng
Vincenzo Cerullo
Akseli Hemminki
Brendan Lee
Baylor College of Medicine · USUniversity of Helsinki · FIHoward Hughes Medical Institute · US

Funding

Improving Safety and Efficacy of Gene Therapy with HDAdR01DK067324 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI NG, PHILIP · 2005 to 2013
$3.5M
In vivo function and tolerance to Factor VIII variantsR01HL087836 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI LEE, BRENDAN · 2008 to 2011
$1.5M
Blocking the immune response to HDAd for Hemophilia A gene therapyR00HL098692 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI SUZUKI, MASATAKA · 2013 to 2015
$738k
Blocking the immune response to Helper-dependent adenovirus vector for HemophiliaK99HL098692 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI SUZUKI, MASATAKA · 2010 to 2011
$164k
NHLBI NIH HHS K99HL098692NHLBI NIH HHS R00 HL098692NHLBI NIH HHS R01HL87836NIDDK NIH HHS R01DK067324
6 · The paper itself

Abstract

Skeletal muscle represents an attractive target tissue for adenoviral gene therapy to treat muscle disorders and as a production platform for systemic expression of therapeutic proteins. However, adenovirus serotype 5 vectors do not efficiently transduce adult muscle tissue. Here we evaluated whether capsid modifications on adenoviral vectors could improve transduction in mature murine muscle tissue. First-generation and helper-dependent serotype 5 adenoviral vectors featuring the serotype 3 knob (5/3) showed significantly increased transduction of skeletal muscle after intramuscular injection in adult mice. Furthermore, we showed that full-length dystrophin could be more efficiently transferred to muscles of mdx mice using a 5/3-modified helper-dependent adenoviral vector. In contrast to first-generation vectors, helper-dependent adenoviral vectors mediated stable marker gene expression for at least 1 year after intramuscular injection. In conclusion, 5/3 capsid-modified helper-dependent adenoviral vectors show enhanced transduction in adult murine muscle tissue and mediate long-term gene expression, suggesting the suitability of these vectors for muscle-directed gene therapy.

Indexed as

Genetic TherapyAdenoviridaeAnimalsCapsidGene ExpressionGenetic VectorsMiceMice, Inbred C57BLMice, Inbred mdxMuscle, SkeletalOrgan SpecificityTime Factors

Identifiers

PMID22888960
PMCPMC3472516
OpenAlexW2112564955

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.