Evidence map›Paper›PMID 33535527›Full record

ReviewJournal of clinical medicine2021

Advances and Obstacles in Homology-Mediated Gene Editing of Hematopoietic Stem Cells.

Christi T Salisbury-Ruf, Andre Larochelle

Open access · goldAbstract readReview
In one paragraph

Review in Journal of clinical medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
1.0field-weighted citation impact, top 26% 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

12 citing papers in PubMed, 13 citations in OpenAlex.

  1. Article
  2. T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  3. Article
  4. Review
  5. Review
  6. Gene Editing in Hematopoietic Stem Cells.Advances in experimental medicine and biology · 2023
    Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. Review
  12. 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

2 authors at 1 institution in 1 country.

Christi T Salisbury-RufCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Andre LarochelleCellular and Molecular Therapeutics Branch, National Heart, Lung and Blood Institute (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.ORCID 0000-0003-0685-2186
National Institutes of Health · US

Funding

Regenerative Therapies for Inherited Blood Disorders-Gene therapyZIAHL006172 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2013 to 2025
$13.0M
Regenerative Therapies for Inherited Blood Disorders-iPSC differentiationZIAHL006217 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2016 to 2025
$8.5M
Regenerative Therapies for Inherited Blood Disorders-HSC expansionZIAHL006218 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI LAROCHELLE, ANDRE · 2016 to 2022
$3.2M
NIH HHS Z99 HL999999
6 · The paper itself

Abstract

Homology-directed gene editing of hematopoietic stem and progenitor cells (HSPCs) is a promising strategy for the treatment of inherited blood disorders, obviating many of the limitations associated with viral vector-mediated gene therapies. The use of CRISPR/Cas9 or other programmable nucleases and improved methods of homology template delivery have enabled precise ex vivo gene editing. These transformative advances have also highlighted technical challenges to achieve high-efficiency gene editing in HSPCs for therapeutic applications. In this review, we discuss recent pre-clinical investigations utilizing homology-mediated gene editing in HSPCs and highlight various strategies to improve editing efficiency in these cells.

Indexed as

AAV6CRISPR-Cas9gene editinghematopoietic stem and progenitor cellshomology-directed repair

Identifiers

PMID33535527
PMCPMC7867106
OpenAlexW3127689232

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.