Evidence map›Paper›PMID 35934593›Full record

ReviewTrends in genetics : TIG2022

Effective therapies for sickle cell disease: are we there yet?

Merlin Crossley, Georgios E Christakopoulos, Mitchell J Weiss

Abstract readReview
In one paragraph

Review in Trends in genetics : TIG, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Article
  2. Article
  3. Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease.Cardiovascular & hematological disorders drug targets · 2026
    Review
  4. Mechanisms of Globin Gene Regulation in Mammals.Annual review of genetics · 2025
    Review
  5. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  6. Review
  7. Treatment of sickle cell disease - options and perspective.American journal of blood research · 2023
    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.

Merlin CrossleySchool of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, Australia 2052. Electronic address: m.crossley@unsw.edu.au.
Georgios E ChristakopoulosDepartment of Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Mitchell J WeissDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

Funding

VIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI WEISS, MITCHELL J · 1994 to 2022
$43.6M
NHLBI NIH HHS P01 HL053749
6 · The paper itself

Abstract

Sickle cell disease (SCD) is a common genetic blood disorder associated with acute and chronic pain, progressive multiorgan damage, and early mortality. Recent advances in technologies to manipulate the human genome, a century of research and the development of techniques enabling the isolation, efficient genetic modification, and reimplantation of autologous patient hematopoietic stem cells (HSCs), mean that curing most patients with SCD could soon be a reality in wealthy countries. In parallel, ongoing research is pursuing more facile treatments, such as in-vivo-delivered genetic therapies and new drugs that can eventually be administered in low- and middle-income countries where most SCD patients reside.

Indexed as

Anemia, Sickle CellHematopoietic Stem Cell TransplantationGene EditingGenetic TherapyHematopoietic Stem CellsHumansbase editingCRISPRgene editinggene therapysickle cell diseaseβ-hemoglobinopathiesβ-thalassemia

Identifiers

PMID35934593
PMCPMC9837857

What OpenQuestion holds

Textmetadata
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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.