Evidence map›Paper›PMID 38062734›Full record

ArticleHuman gene therapy2024

Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67

Thomas E Whittaker, Shefta E Moula, Sameer Bahal, Faris Ghalib Bakri, Wail Ahmad Hayajneh, Ammar Khaled Daoud, Asma Naseem, Alessia Cavazza, Adrian J Thrasher, Giorgia Santilli

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
0.8field-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

4 citing papers in PubMed, 5 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. 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

10 authors at 4 institutions in 3 countries.

Thomas E WhittakerInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Shefta E MoulaInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Sameer BahalInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Faris Ghalib BakriDivision of Infectious Diseases, Department of Medicine, Jordan University Hospital, Amman, Jordan.
Wail Ahmad HayajnehDivision of Infectious Diseases, Department of Pediatrics, Jordan University of Science & Technology, Irbid, Jordan.
Ammar Khaled DaoudDivision of Immunology, Department of Internal Medicine, Jordan University of Science & Technology, Irbid, Jordan.
Asma NaseemInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Alessia CavazzaInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.ORCID 0000-0002-4735-2121
Adrian J ThrasherInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.ORCID 0000-0002-6097-6115
Giorgia SantilliInfection, Immunity, and Inflammation Research and Teaching Department, UCL, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.ORCID 0000-0003-1776-1984
Institute of Infection and Immunity · CAJordan University of Science and Technology · JOGreat Ormond Street Hospital · GBUniversity of Jordan · JO

Funding

Medical Research Council MR/S021930/1Wellcome Trust 104807Wellcome Trust 217112/Z/19/Z
6 · The paper itself

Abstract

Replacing a faulty gene with a correct copy has become a viable therapeutic option as a result of recent progress in gene editing protocols. Targeted integration of therapeutic genes in hematopoietic stem cells has been achieved for multiple genes using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 system and Adeno-Associated Virus (AAV) to carry a donor template. Although this is a promising strategy to correct genetic blood disorders, it is associated with toxicity and loss of function in CD34

Indexed as

Granulomatous Disease, ChronicAntigens, CD34CRISPR-Cas SystemsGene EditingGenetic TherapyHematopoietic Stem CellsHumansAntigens, CD34AAVCGDgene editinghomology-directed repairresponse surface methodology

Identifiers

PMID38062734
PMCPMC7615834
OpenAlexW4389486522

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.