Evidence map›Paper›PMID 41396678›Full record

ReviewBlood advances2025

Challenges and limitations of mobilization and stem cell collection for gene therapy of sickle cell disease.

Yvette C Tanhehco, Suzanne Thibodeaux, Patricia A Shi, Stella T Chou, Shannon A Kelly, Grace Fortes Monis, Carly Newton, Kamille West-Mitchell, Yan Zheng, Christine Fernandez and 5 more

Abstract readReview
In one paragraph

Review in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

15 authors.

Yvette C TanhehcoDepartment of Pathology and Cell Biology, Columbia University Irving Medical Center, New York, NY.ORCID 0000-0002-7800-3119
Suzanne ThibodeauxDepartment of Pathology and Immunology, Washington University School of Medicine, St. Louis, MO.ORCID 0000-0002-9881-703X
Patricia A ShiClinical Research in Sickle Cell Disease, Lindsley F. Kimball Research Institute, New York Blood Center, New York, NY.ORCID 0000-0002-7954-0055
Stella T ChouDepartment of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, PA.ORCID 0000-0003-4333-6965
Shannon A KellyDepartment of Pediatrics, University of California San Francisco Benioff Children's Hospital Oakland, Oakland, CA.
Grace Fortes MonisDepartment of Pathology and Laboratory Medicine, University of California Davis Health, Sacramento, CA.
Carly NewtonApheresis Clinical Marketing, Terumo Blood and Cell Technologies, Lakewood, CO.
Kamille West-MitchellDepartment of Transfusion Medicine, National Institutes of Health, Bethesda, MA.ORCID 0000-0001-8152-804X
Yan ZhengDepartment of Pathology, St. Jude Children's Research Hospital, Memphis, TN.ORCID 0000-0001-7254-7564
Christine FernandezCRISPR Therapeutics, Boston, MA.
Christina AndersonCRISPR Therapeutics, Boston, MA.
Kimberly ChingDepartment of Laboratory Medicine, Boston Children's Hospital, Boston, MA.
Victoria Coleman-CowgerThe Emmes Company, Rockville, MD.
Adrienne ShapiroAxis Advocacy, Pasadena, CA.
John P ManisDepartment of Laboratory Medicine, Boston Children's Hospital, Boston, MA.

Funding

Novel Strategies to Improve Blood Transfusion PracticeP01HL158688 · NHLBI · BRIGHAM AND WOMEN'S HOSPITAL · PI Hongbo R Luo · 2022 to 2026
$12.9M
TRANSFUSION BIOLOGY AND MEDICINET32HL066987 · NHLBI · CHILDREN'S HOSPITAL BOSTON · PI JOHN P MANIS · 2001 to 2026
$10.2M
NHLBI NIH HHS P01 HL158688NHLBI NIH HHS T32 HL066987
6 · The paper itself

Abstract

abstractSickle cell disease (SCD) is a hemolytic anemia that afflicts millions of people worldwide and continues to carry high morbidity and reduced life expectancy. Allogeneic hematopoietic stem cell transplantation was the only standard-of-care option for cure before the US Food and Drug Administration's approval of 2 cellular drugs. Transfusion medicine plays a pivotal role in supporting patients through mobilization and apheresis collection of peripheral blood stem cells and through transplant using standard or exchange transfusion strategies. Despite these advances, obtaining sufficient cells to generate a cellular gene therapy product and lack of standardized protocols that describe the optimal preparative regimen, mobilization, and collection of stem cells present a significant barrier to success. Our working group, convened as part of the National Heart, Lung, and Blood Institute-funded Cure Sickle Cell Initiative, sought to identify gaps in our understanding of these processes to improve advanced cell therapies for SCD.

Indexed as

Anemia, Sickle CellGenetic TherapyHematopoietic Stem Cell MobilizationHematopoietic Stem Cell TransplantationHumans

Identifiers

PMID41396678
PMCPMC12767821

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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.