Related Experiment Video
Updated: Apr 3, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Cellular engineering and therapy in combination with cord blood allografting in pediatric recipients
M S Cairo1,2,3,4,5, N Tarek6, D A Lee6
1Department of Pediatrics, Maria Fareri Children's Hospital at Westchester Medical Center, New York Medical College, Valhalla, NY, USA.
Insights
Cord blood (CB) transplantation faces challenges like delayed immune recovery. Cellular engineering strategies, including ex vivo expansion and novel cell sources, show promise in overcoming these limitations for better patient outcomes.
Area of Science:
- Hematology
- Immunology
- Cellular Therapy
Background:
- Cord blood (CB) transplantation is a vital source of hematopoietic progenitor cells for pediatric allogeneic stem cell transplants.
- Current limitations include delayed hematopoietic reconstitution, graft failure, and slow immune recovery, increasing transplant-related mortality.
- Cellular engineering offers potential solutions to these challenges.
Purpose of the Study:
- To review recent experimental approaches in cellular engineering for ex vivo cord blood expansion.
- To summarize findings on strategies aimed at improving hematopoietic and immune reconstitution after unrelated CB transplantation.
Main Methods:
- Review of experimental approaches including ex vivo CB expansion with Notch1 ligand Delta 1.
- Investigation of mesenchymal progenitor cells, placenta-derived stem cells, and CB-derived natural killer cells.
- Analysis of preliminary results from these cellular strategies.
Main Results:
- Early findings suggest experimental cellular strategies may mitigate primary graft failure.
- These approaches may also address delays in hematopoietic and immune reconstitution.
- Preliminary data indicate potential improvements in outcomes following unrelated CB transplantation.
Conclusions:
- Experimental cellular strategies show promise in addressing key limitations of cord blood transplantation.
- Further research into ex vivo expansion and novel cell sources is warranted.
- These advancements could improve the safety and efficacy of pediatric stem cell transplantation.
Abstract:
Cord blood (CB) transplantation is an alternate source of human hematopoietic progenitor cells for allogeneic stem cell transplantation in children and adolescents with both malignant and nonmalignant diseases. Current limitations included delay in hematopoietic reconstitution, increased incidence of primary graft failure and slow cellular immunoreconstitution. These limitations lead to a significant increase in primary graft failure, infectious complications and increased transplant-related mortality. There is a number of experimental approaches currently under investigation including cellular engineering to circumvent these limitations. In this review, we summarize the recent findings of utilizing ex vivo CB expansion with Notch1 ligand Delta 1, mesenchymal progenitor cells, the use of human placenta-derived stem cells and CB-derived natural killer cells. Early and preliminary results suggest some of these experimental cellular strategies may in part ameliorate the incidence of primary graft failure, delays in hematopoietic reconstitution and/or slowness in cellular immune reconstitution following unrelated CB transplantation.
More Related Videos
11:18Expanding Cytotoxic T Lymphocytes from Umbilical Cord Blood that Target Cytomegalovirus, Epstein-Barr Virus, and Adenovirus
Published on: May 7, 2012
09:29Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood
Published on: February 1, 2013
Related Concept Videos
Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
iPS Cell Differentiation
Stem Cell Culture