Evidence map›Paper›PMID 41707137›Full record

Trial reportThe New England journal of medicine2026

Hematopoietic Stem-Cell Gene Therapy for Cystinosis.

Bruce A Barshop, Edward D Ball, Nadine Benador, Doris Trauner, Susan Phillips, Ranjan Dohil, Natalie A Afshari, Sohini Roy, Beatriz Campo Fernandes, Donald Kohn and 10 more

Registry-linked trialAbstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The New England journal of medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03897361 (A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells), which is not on this map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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.

NCT03897361 phase1 / phase2completednot on this map

A Phase 1/2 Study to Determine Safety and Efficacy of Transplantation With Autologous Human CD34+ Hematopoietic Stem Cells (HSC) From Mobilized Peripheral Blood Stem Cells (PBSC) of Patients With Cystinosis Modified by Ex Vivo Transduction Using pCCL-CTNS or pCDY.EFS.CTNS.T260I Lentiviral Vector and Will Include Transduction Enhancer When Required During Manufacturing

TypeinterventionalSponsorUniversity of California, San DiegoRan2019 to 2024Enrolled7ConditionsLysosomal Storage Diseases, CystinosisArmsCTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Article
  5. Pediatric Nephrology in Clinical Practice.Indian journal of pediatrics · 2026
    Article
  6. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

20 authors.

Bruce A BarshopDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
Edward D BallDepartment of Medicine, Division of Blood and Marrow Transplantation, University of California, San Diego, La Jolla.
Nadine BenadorDepartment of Pediatrics, Division of Nephrology, University of California, San Diego, La Jolla.
Doris TraunerDepartment of Neurosciences, University of California, San Diego, La Jolla.
Susan PhillipsRady Children's Hospital, San Diego, CA.
Ranjan DohilRady Children's Hospital, San Diego, CA.
Natalie A AfshariDepartment of Ophthalmology, University of California, San Diego, La Jolla.
Sohini RoyDepartment of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles.
Beatriz Campo FernandesDepartment of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles.
Donald KohnDepartment of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles.
Katayoon ShayanRady Children's Hospital, San Diego, CA.
John K EverettDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
Frederic D BushmanDepartment of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia.ORCID 0000-0003-4740-4056
Julian MidgleyDepartment of Pediatrics, University of Calgary, Calgary, AB, Canada.
Hong LiangDepartment of Ophthalmology III, Quinze-Vingts National Ophthalmology Hospital, DHU Sight Restore, INSERM-DHOS CIC, Paris.
Anne SawyersDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
Jon A GangoitiDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
Maulik PanchalDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
Imama AhmedDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.
Stephanie CherquiDepartment of Pediatrics, Division of Genetics, University of California, San Diego, La Jolla.ORCID 0000-0003-1240-5219

Funding

Microgial contribution and therapeutic potential in Friedreich's ataxiaR01NS135162 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Stephanie Cherqui, Nicole Gabriele Coufal · 2024 to 2026
$1.8M
California Institute for Regenerative Medicine CLIN2-11478NINDS NIH HHS R01 NS135162Novartis 30327993
6 · The paper itself

Abstract

backgroundCystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in

methodsIn this phase 1-2, open-label, ongoing clinical study, we performed a preliminary assessment of CTNS-RD-04, which consists of autologous CD34+ cells transduced with lentiviral vectors carrying

resultsSix participants (20 to 46 years of age) received CTNS-RD-04 and were followed for 29 to 63 months. CTNS-RD-04 doses ranged from 3.63×10

conclusionsIn this small study, CTNS-RD-04, an ex vivo gene therapy for cystinosis, had adverse effects that were largely consistent with the myeloablative regimen and underlying disease profile. White-cell cystine levels decreased after therapy. (Funded by the California Institute for Regenerative Medicine and others; ClinicalTrials.gov number, NCT03897361.).

Indexed as

Amino Acid Transport Systems, NeutralCystinosisGenetic TherapyHematopoietic Stem Cell TransplantationAdultCysteamineCystineCystine Depleting AgentsDisease ProgressionFemaleFollow-Up StudiesGenetic VectorsHumansLentivirusMaleMiddle AgedAmino Acid Transport Systems, NeutralCTNS protein, humanCysteamineCystineCystine Depleting AgentsMyeloablative Agonists

Identifiers

PMID41707137
PMCPMC12919641

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

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.