Evidence map›Paper›PMID 26333349›Full record

ArticleGene therapy2015

Development of operational immunologic tolerance with neonatal gene transfer in nonhuman primates: preliminary studies.

D S Tai, C Hu, C C I Lee, M Martinez, G Cantero, E H Kim, A F Tarantal, G S Lipshutz

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

Article in Gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
0.7field-weighted citation impact, top 24% 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

9 citing papers in PubMed, 9 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Durability of transgene expression after rAAV gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    Review
  5. Dosing and Re-Administration of Lentiviral Vector forMolecular therapy. Methods & clinical development · 2020
    Article
  6. Prenatal treatment path for angelman syndrome and other neurodevelopmental disorders.Autism research : official journal of the International Society for Autism Research · 2020
    Review
  7. Article
  8. Article
  9. Rescue of the Functional Alterations of Motor Cortical Circuits in Arginase Deficiency by Neonatal Gene Therapy.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2016
    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

8 authors at 2 institutions in 1 country.

D S TaiDepartment of Surgery, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
C HuDepartment of Surgery, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
C C I LeeCalifornia National Primate Research Center and Departments of Pediatrics and Cell Biology and Human Anatomy, University of California, Davis, CA, USA.
M MartinezCalifornia National Primate Research Center and Departments of Pediatrics and Cell Biology and Human Anatomy, University of California, Davis, CA, USA.
G CanteroDepartment of Surgery, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
E H KimDepartment of Surgery, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
A F TarantalCalifornia National Primate Research Center and Departments of Pediatrics and Cell Biology and Human Anatomy, University of California, Davis, CA, USA.
G S LipshutzDepartment of Surgery, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
University of California, Los Angeles · USUniversity of California, Davis · US

Funding

National Institute on Aging (NIA) ColonyP51OD011107 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI Simon J. Atkinson · 2012 to 2026
$191.5M
Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood DiseasesR24HL085794 · NHLBI · UNIVERSITY OF CALIFORNIA AT DAVIS · PI TARANTAL, ALICE F · 2006 to 2016
$9.7M
UCLA Medical Genetics Training ProgramT32GM008243 · NIGMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Julian Martinez-Agosto, STANLEY F. NELSON · 1987 to 2026
$6.8M
Pathophysiology and Gene Replacement Strategies for Arginase DeficiencyR01NS071076 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2010 to 2014
$1.7M
The Immune Response to Viral Vector and Transgenes in the FetusK08HD057555 · NICHD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2007 to 2011
$558k
Immunologic Aspects of In Utero or Neonatal AAV-Based Gene TherapyR56AI108826 · NIAID · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S, TARANTAL, ALICE F · 2014 to 2014
$513k
Ultrasound Imaging for Nonhuman Primate Translational ResearchS10OD016261 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI TARANTAL, ALICE F · 2013 to 2013
$203k
NHLBI NIH HHS HL85794NHLBI NIH HHS R24 HL085794NIAID NIH HHS AI108826-01NIAID NIH HHS R56 AI108826NICHD NIH HHS HD057555NICHD NIH HHS K08 HD057555NIGMS NIH HHS T32 GM008243NIH HHS OD011107NIH HHS P51 OD011107NIH HHS S10 OD016261NINDS NIH HHS NS071076NINDS NIH HHS NS071076-04S1NINDS NIH HHS R01 NS071076
6 · The paper itself

Abstract

Achieving persistent expression is a prerequisite for effective genetic therapies for inherited disorders. These proof-of-concept studies focused on adeno-associated virus (AAV) administration to newborn monkeys. Serotype rh10 AAV expressing ovalbumin and green fluorescent protein (GFP) was administered intravenously at birth and compared with vehicle controls. At 4 months postnatal age, a second injection was administered intramuscularly, followed by vaccination at 1 year of age with ovalbumin and GFP. Ovalbumin was highest 2 weeks post administration in the treated monkey, which declined but remained detectable thereafter; controls demonstrated no expression. Long-term AAV genome copies were present in myocytes. At 4 weeks, neutralizing antibodies to rh10 were present in the experimental animal only. With AAV9 administration at 4 months, controls showed transient ovalbumin expression that disappeared with the development of strong anti-ovalbumin and anti-GFP antibodies. In contrast, increased and maintained ovalbumin expression was noted in the monkey administered AAV at birth, without antibody development. After vaccination, the experimental monkey maintained levels of ovalbumin without antibodies, whereas controls demonstrated high levels of antibodies. These preliminary studies suggest that newborn AAV administration expressing secreted and intracellular xenogenic proteins may result in persistent expression in muscle, and subsequent vector administration can result in augmented expression without humoral immune responses.

Indexed as

Gene Transfer TechniquesAnimalsAnimals, NewbornAntibodies, HeterophileAntibodies, NeutralizingDependovirusFemaleGenetic TherapyGenetic VectorsImmune ToleranceImmunity, HumoralMacaca mulattaOvalbuminPilot ProjectsAntibodies, HeterophileAntibodies, NeutralizingOvalbumin

Identifiers

PMID26333349
PMCPMC5483994
OpenAlexW1210978933

What OpenQuestion holds

Textmetadata
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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.