Evidence map›Paper›PMID 32506752›Full record

ReviewBritish journal of haematology2021

CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside.

Olivier Humbert, Clare Samuelson, Hans-Peter Kiem

Open access · greenAbstract readReview
In one paragraph

Review in British journal of haematology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.4field-weighted citation impact, top 40% 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

10 citing papers in PubMed, 17 citations in OpenAlex.

  1. Review
  2. Review
  3. Advances in Cell and Gene Therapy for Rare Disease Treatment.International journal of stem cells · 2025
    Review
  4. Review
  5. Review
  6. Therapeutic precision, potency and promise.Nature biomedical engineering · 2025
    Article
  7. Review
  8. Review
  9. Review
  10. 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

3 authors at 2 institutions in 1 country.

Olivier HumbertFred Hutchinson Cancer Research Center, Seattle, WA, USA.
Clare SamuelsonFred Hutchinson Cancer Research Center, Seattle, WA, USA.
Hans-Peter KiemFred Hutchinson Cancer Research Center, Seattle, WA, USA.ORCID 0000-0001-5949-4947
Fred Hutch Cancer Center · USUniversity of Washington · US

Funding

Cell and Gene Therapy for HIV CureUM1AI126623 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI JEROME, KEITH R, KIEM, HANS-PETER · 2016 to 2020
$24.2M
ZFN-Modified Stem Cells for HIV EradicationU19AI096111 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI DE ROSA, STEPHEN C · 2011 to 2015
$20.8M
Novel Gene Editing Approaches for HemoglobinopathiesR01HL136135 · NHLBI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI HANS-PETER KIEM · 2017 to 2026
$7.7M
Targeting a Novel Hematopoietic Stem Cell Population in Non-Human Primates for Effective and Sustained Gene TherapyR01AI135953 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI KIEM, HANS-PETER · 2018 to 2022
$4.5M
Nongenotoxic conditioning for gene therapy and allogeneic transplantation in Fanconi anemiaR01HL147324 · NHLBI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Bruce R Blazar, HANS-PETER KIEM · 2020 to 2026
$4.1M
NHLBI NIH HHS R01 HL136135NHLBI NIH HHS R01 HL147324NIAID NIH HHS R01 AI135953NIAID NIH HHS U19 AI096111NIAID NIH HHS UM1 AI126623
6 · The paper itself

Abstract

Genome editing therapies represent a significant advancement in next-generation, precision medicine for the management of haematological diseases, and CRISPR/Cas9 has to date been the most successful implementation platform. From discovery in bacteria and archaea over three decades ago, through intensive basic research and pre-clinical development phases involving the modification of therapeutically relevant cell types, CRISPR/Cas9 genome editing is now being investigated in ongoing clinic trials. Despite the widespread enthusiasm brought by this new technology, significant challenges remain before genome editing can be routinely recommended and implemented in the clinic. These include risks of genotoxicity resulting from off-target DNA cleavage or chromosomal rearrangement, and suboptimal efficacy of homology-directed repair editing strategies, which thus limit therapeutic options. Practical hurdles such as high costs and inaccessibility to patients outside specialised centres must also be addressed. Future improvements in this rapidly developing field should circumvent current limitations with novel editing platforms and with the simplification of clinical protocols using in vivo delivery of editing reagents.

Indexed as

CRISPR-Cas SystemsAnimalsGene EditingGenetic TherapyHematologic DiseasesHumans

Identifiers

PMID32506752
PMCPMC8118330
OpenAlexW3033405009

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.