Evidence map›Paper›PMID 41235102›Full record

ReviewCurrent genomics2025

Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives.

Sundus Khawaja, Raja Hussain Ali, Ishtiaq Ahmed, Muhammad Umair

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Current genomics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Preclinical efficacy of a gene therapy forMolecular therapy. Advances · 2026
    Article
  2. Review
  3. Novel advanced patient-derivedFrontiers in neurology · 2026
    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

4 authors.

Sundus KhawajaDepartment of Biotechnology, University of Azad Jammu and Kashmir, Muzaffarabad, Pakistan.
Raja Hussain AliDepartment of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Teaching Hospital, Boston, Massachusetts, USA.
Ishtiaq AhmedInstitute of Biochemistry and Biotechnology, Pir Mehr Ali Shah Arid Agriculture University Rawalpindi 46000, Pakistan.
Muhammad UmairMedical Genomics Research Department, King Abdullah International Medical Research Center (KAIMRC), King Saud Bin Abdulaziz University for Health Sciences, Ministry of National Guard Health Affairs (MNGH), Riyadh, Saudi Arabia.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Rare genetic disorders collectively affect millions of individuals worldwide, presenting a significant clinical and research challenge due to the diversity and complexity of the underlying mutations. Current treatment options are often limited, focusing on symptom management rather than addressing the root genetic causes. This review article aims to provide a perspective on the evolving field of gene therapy for rare genetic disorders, emphasizing recent advancements, current challenges, and future directions. A comprehensive review of recent advancements in gene therapy for rare genetic disorders was conducted, focusing on therapeutic strategies, delivery systems, and clinical outcomes. Key examples, such as the use of viral vectors and gene-editing technologies (

Indexed as

ex vivo gene therapyGene therapyin vivo gene therapyrare genetic disordersZolgensma

Identifiers

PMID41235102
PMCPMC12606653

What Socratic 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 Socratic graph.