Evidence map›Paper›PMID 42761173›Full record

ReviewMolecular therapy. Nucleic acids2026

Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons.

Zhenghe Chen, Wei Wang, Youlan Wu, Yawen Qiang, Weisheng Cheng, Fang Liu

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

6 authors.

Zhenghe ChenDepartment of Clinical Laboratory, the First Affiliated Hospital of Anhui Medical University, No. 218 Jixi Road, Hefei, Anhui 230022, China.
Wei WangDepartment of Pathology, the First Affiliated Hospital of Anhui Medical University, No. 218 Jixi Road, Hefei, Anhui 230022, China.
Youlan WuPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, No. 218 Jixi Road, Hefei, Anhui 230022, China.
Yawen QiangPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, No. 218 Jixi Road, Hefei, Anhui 230022, China.
Weisheng ChengPrenatal Diagnosis Center, Department of Obstetrics and Gynecology, the First Affiliated Hospital of Anhui Medical University, No. 218 Jixi Road, Hefei, Anhui 230022, China.
Fang LiuDepartment of Clinical Laboratory, the First Affiliated Hospital of Anhui Medical University, No. 218 Jixi Road, Hefei, Anhui 230022, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hereditary hematological disorders, including sickle cell disease, β-thalassemia, and hemophilia, are severe monogenic diseases that impose substantial morbidity and lifelong treatment burdens. Conventional therapies are largely supportive and rarely curative, whereas gene therapy is increasingly transforming the therapeutic landscape by addressing the underlying genetic defects. This review summarizes the major gene therapy strategies currently being developed for hereditary hematological disorders, with a particular focus on gene addition, gene editing, and gene silencing, as well as

Indexed as

gene therapyhemophiliahereditary hematological disordersMT: Oligonucleotides: Therapies and Applicationssickle cell diseasethalassemia

Identifiers

PMID42761173
PMCPMC13586514

What OpenQuestion holds

Textmetadata
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Registered trials

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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.