Evidence map›Paper›PMID 24925206›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2014

Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys.

Denise Carbonaro Sarracino, Alice F Tarantal, C Chang I Lee, Michele Martinez, Xiangyang Jin, Xiaoyan Wang, Cinnamon L Hardee, Sabine Geiger, Christoph A Kahl, Donald B Kohn

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, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
0.4field-weighted citation impact, top 36% 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

7 citing papers in PubMed, 8 citations in OpenAlex.

  1. Article
  2. Article
  3. Gene delivery using AAV8Molecular therapy. Methods & clinical development · 2021
    Article
  4. Review
  5. Dosing and Re-Administration of Lentiviral Vector forMolecular therapy. Methods & clinical development · 2020
    Article
  6. Review
  7. Targeted approaches to induce immune tolerance for Pompe disease therapy.Molecular therapy. Methods & clinical development · 2016
    Review
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

10 authors at 3 institutions in 1 country.

Denise Carbonaro SarracinoDepartment of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California, USA.
Alice F Tarantal1] Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, University of California, Davis, California USA [2] Departments of Pediatrics and Cell Biology and Human Anatomy, University of California, Davis, CA, USA.
C Chang I LeeCenter for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, University of California, Davis, California USA.
Michele MartinezCenter for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, University of California, Davis, California USA.
Xiangyang JinDepartment of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California, USA.
Xiaoyan WangDepartment of General Internal Medicine and Health Services Research, University of California, Los Angeles California, USA.
Cinnamon L HardeeDepartment of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California, USA.
Sabine GeigerDepartment of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California, USA.
Christoph A Kahl1] Division of Research Immunology/BMT, Children's Hospital Los Angeles, Los Angeles, California, USA [2] Current address: Oregon National Primate Research Center, Oregon Health and Science University, Beaverton, Oregon, USA.
Donald B Kohn1] Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California, USA [2] Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, California, USA.
University of California, Los Angeles · USUniversity of California, Davis · USOregon Health & Science University · US

Funding

National Institute on Aging (NIA) ColonyP51OD011107 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI Simon J. Atkinson · 2012 to 2026
$191.5M
ZONA PELLUCIDA PEPTIDES AS IMMUNOCONTRACEPTIVE ANTIGENSP51RR000169 · NCRR · UNIVERSITY OF CALIFORNIA DAVIS · PI HYDE, DALLAS MELVIN · 1985 to 2011
$126.2M
Transduction of Human Stem and Progenitor CellsP01HL073104 · NHLBI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI KOHN, DONALD B · 2003 to 2013
$18.3M
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
In Vivo ADA Gene Delivery for the Treatment of SCIDR01AI074043 · NIAID · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI KOHN, DONALD B · 2007 to 2011
$2.3M
NCRR NIH HHS P51 RR000169NHLBI NIH HHS HL073104NHLBI NIH HHS HL085794NHLBI NIH HHS P01 HL073104NHLBI NIH HHS R24 HL085794NIAID NIH HHS R01 AI074043NIAID NIH HHS R01-AI074043NIH HHS P51 OD011107
6 · The paper itself

Abstract

Systemic delivery of a lentiviral vector carrying a therapeutic gene represents a new treatment for monogenic disease. Previously, we have shown that transfer of the adenosine deaminase (ADA) cDNA in vivo rescues the lethal phenotype and reconstitutes immune function in ADA-deficient mice. In order to translate this approach to ADA-deficient severe combined immune deficiency patients, neonatal ADA-deficient mice and newborn rhesus monkeys were treated with species-matched and mismatched vectors and pseudotypes. We compared gene delivery by the HIV-1-based vector to murine γ-retroviral vectors pseudotyped with vesicular stomatitis virus-glycoprotein or murine retroviral envelopes in ADA-deficient mice. The vesicular stomatitis virus-glycoprotein pseudotyped lentiviral vectors had the highest titer and resulted in the highest vector copy number in multiple tissues, particularly liver and lung. In monkeys, HIV-1 or simian immunodeficiency virus vectors resulted in similar biodistribution in most tissues including bone marrow, spleen, liver, and lung. Simian immunodeficiency virus pseudotyped with the gibbon ape leukemia virus envelope produced 10- to 30-fold lower titers than the vesicular stomatitis virus-glycoprotein pseudotype, but had a similar tissue biodistribution and similar copy number in blood cells. The relative copy numbers achieved in mice and monkeys were similar when adjusted to the administered dose per kg. These results suggest that this approach can be scaled-up to clinical levels for treatment of ADA-deficient severe combined immune deficiency subjects with suboptimal hematopoietic stem cell transplantation options.

Indexed as

Gene Transfer TechniquesTransduction, GeneticAdenosine DeaminaseAnimalsDisease Models, AnimalFemaleGene ExpressionGene OrderGenetic VectorsHumansLentivirusMacaca mulattaMiceMice, KnockoutRetroviridaeSevere Combined ImmunodeficiencyAdenosine Deaminase

Identifiers

PMID24925206
PMCPMC4428412
OpenAlexW2047379445

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.