Evidence map›Paper›PMID 32253938›Full record

ReviewHuman gene therapy2020

Adeno-Associated Virus and Hematopoietic Stem Cells: The Potential of Adeno-Associated Virus Hematopoietic Stem Cells in Genetic Medicines.

Saswati Chatterjee, Venkatesh Sivanandam, Kamehameha Kai-Min Wong

Abstract readReview
In one paragraph

Review in Human gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
–field-weighted citation impact
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

11 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Treating genetic blood disorders in the era of CRISPR-mediated genome editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  5. Article
  6. Get Spliced: Uniting Alternative Splicing and Arthritis.International journal of molecular sciences · 2024
    Review
  7. Advances in HIV Gene Therapy.International journal of molecular sciences · 2024
    Review
  8. Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  9. Novel vectors and approaches for gene therapy in liver diseases.JHEP reports : innovation in hepatology · 2021
    Review
  10. Review
  11. 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

3 authors.

Saswati ChatterjeeDepartment of Surgery, Beckman Research Institute of City of Hope Medical Center, Duarte, California, USA.
Venkatesh SivanandamDepartment of Surgery, Beckman Research Institute of City of Hope Medical Center, Duarte, California, USA.
Kamehameha Kai-Min WongDepartment of Hematology and Stem Cell Transplantation, City of Hope Medical Center, Duarte, California, USA.

Funding

Transgenic Mouse FacilityP30CA033572 · NCI · CITY OF HOPE/BECKMAN RESEARCH INSTITUTE · PI XIWEI WU · 1985 to 2026
$86.3M
MICROBIAL GENOME SEQUENCING CENTERSN01AI40001 · NIAID · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · PI BIRREN, BRUCE W. · 2007 to 2009
$14.1M
Genetic Modification of Human Hematopoietic Stem Cells with Pseudotyped rAAVR01HL087285 · NHLBI · BECKMAN RESEARCH INSTITUTE/CITY OF HOPE · PI CHATTERJEE, SASWATI · 2008 to 2011
$1.7M
MULTIVALENT AAV VECTORS FOR HIV-1 GENE THERAPYR01AI040001 · NIAID · CITY OF HOPE NATIONAL MEDICAL CENTER · PI CHATTERJEE, SASWATI · 1996 to 1998
–
NCI NIH HHS P30 CA033572NHLBI NIH HHS R01 HL087285NIAID NIH HHS N01AI40001
6 · The paper itself

Abstract

Adeno-associated virus (AAV)-based vectors have transformed into powerful elements of genetic medicine with proven therapeutic efficacy and a good safety profile. Over the years, efforts to transduce hematopoietic stem cells (HSCs) with AAV2 vectors have, however, been challenging. While there was evidence that AAV2 delivered vector genomes to primitive, quiescent, multipotential, self-renewing,

Indexed as

Gene EditingGenetic TherapyAnimalsDependovirusHematopoietic Stem CellsHumansLeukodystrophy, MetachromaticPhenylketonuriasTransduction, GeneticTransgenesAAVAAVHSCgene editinggene therapyhematopoietic stem cellsPKU

Identifiers

PMID32253938
PMCPMC7232699

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.