Evidence map›Paper›PMID 35773053›Full record

ReviewHematology/oncology clinics of North America2022

Evolution of Gene Therapy, Historical Perspective.

Harry L Malech, Elizabeth K Garabedian, Matthew M Hsieh

Open access · greenAbstract readReview
In one paragraph

Review in Hematology/oncology clinics of North America, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
3.6field-weighted citation impact, top 6% 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

15 citing papers in PubMed, 41 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Landscape ofMolecular therapy. Methods & clinical development · 2025
    Article
  5. Review
  6. Review
  7. Article
  8. Review
  9. Review
  10. Clinical hematopoietic stem cell-based gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  11. Article
  12. Article
  13. Article
  14. Review
  15. 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 at 3 institutions in 1 country.

Harry L MalechGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, 10 Center Drive, MSC1456, Bldg 10, Rm 5-3750, Bethesda, MD 20892-1456, USA. Electronic address: hmalech@niaid.nih.gov.
Elizabeth K GarabedianNational Human Genome Research Institute, NIH, 10 Center Dr, MSC1611, Bldg 10, Rm 10C-103, Bethesda, MD 20892-1611, USA.
Matthew M HsiehNational Heart, Lung, and Blood Institute, NIH, 10 Center Drive, MSC1812, Bldg 10, Rm 9N119, Bethesda, MD 20892-1812, USA.
National Heart Lung and Blood Institute · USNational Human Genome Research Institute · USNational Institute of Allergy and Infectious Diseases · US

Funding

Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
PERIPHERAL BLOOD PROGENITORS-TARGET FOR GENE TRANSFERZ01AI000644 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 1992 to 2008
$4.3M
GENE THERAPY FOR IMMUNE DEFICIENCIESZ01AI000645 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI DE RAVIN, SUK · 1992 to 2008
$873k
Intramural NIH HHS Z01 AI000644Intramural NIH HHS Z01 AI000645
6 · The paper itself

Abstract

The earliest conceptual history of gene therapy began with the recognition of DNA as the transforming substance capable of changing the phenotypic character of a bacterium and then as the carrier of the genomic code. Early studies of oncogenic viruses that could insert into the mammalian genome led to the concept that these same viruses might be engineered to carry new genetic material into mammalian cells, including human hematopoietic stem cells (HSC). In addition to properly engineered vectors capable of efficient safe transduction of HSC, successful gene therapy required the development of efficient materials, methods, and equipment to procure, purify, and culture HSC. Increased understanding of the preparative conditioning of patients was needed to optimize the engraftment of genetically modified HSC. Testing concepts in pivotal clinical trials to assess the efficacy and determine the cause of adverse events has advanced the efficiency and safety of gene therapy. This article is a historical overview of the separate threads of discovery that joined together to comprise our current state of gene therapy targeting HSC.

Indexed as

Genetic VectorsHematopoietic Stem Cell TransplantationAnimalsGenetic TherapyHematopoietic Stem CellsHumansLentivirusMammalsTransduction, GeneticApheresisCD34+ HSCGamma retrovirus vectorGene editingHematopoietic stem cellsInsertional mutagenesisLentiviral vectorTransduction

Identifiers

PMID35773053
PMCPMC9296588
OpenAlexW4283579744

What OpenQuestion holds

Textmetadata
LicenceTDM
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.