Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.

Hematol Oncol Clin North Am

Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 6N240A, Bethesda, MD 20892, USA. Electronic address:

Published: December 2022

AI Article Synopsis

  • Curative therapies for sickle cell disease (SCD) include allogeneic HCT, gene therapy, and gene editing, but there's a lack of data to compare their effectiveness.
  • There's a need for new approaches to reduce risks like graft rejection and graft-versus-host disease in haploidentical HCT.
  • Recent studies from the past 5 years are reviewed to explore advancements in these curative therapies for SCD.

Article Abstract

Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might facilitate selecting one curative therapy over another are unavailable. New strategies to decrease graft rejection and graft-versus-host disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene therapy and gene editing also has limitations. Herein, we review recent studies on curative therapies for SCD in the past 5 years.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9681017PMC
http://dx.doi.org/10.1016/j.hoc.2022.06.007DOI Listing

Publication Analysis

Top Keywords

gene therapy
12
curative therapies
8
therapy gene
8
gene editing
8
gene
5
allogeneic transplant
4
transplant gene
4
therapy
4
therapy evolving
4
evolving cure
4

Similar Publications

Targeting RNA splicing modulation: new perspectives for anticancer strategy?

J Exp Clin Cancer Res

January 2025

Department of Pharmacology, School of Pharmacy, China Medical University, No.77 Puhe Road, Shenyang North New Area, Shenyang, Liaoning Province, 110122, P. R. China.

The excision of introns from pre-mRNA is a crucial process in the expression of the majority of genes. Alternative splicing allows a single gene to generate diverse mRNA and protein products. Aberrant RNA splicing is recognized as a molecular characteristic present in almost all types of tumors.

View Article and Find Full Text PDF

Background: Vibrio parahaemolyticus is a marine bacterium causing seafood-associated gastrointestinal illness in humans and acute hepatopancreatic necrosis disease (AHPND) in shrimp. Bacteriophages have emerged as promising biocontrol agents against V. parahaemolyticus.

View Article and Find Full Text PDF

High-dose dual therapy for Helicobacter pylori eradication inducing less impact on the gut microbiota.

Gut Pathog

January 2025

Division of Gastroenterology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, 1095 Jiefang Avenue, Wuhan, 430030, China.

Background: Helicobacter pylori (H. pylori) eradication regimens may have different effects on the gut microbiota. Few studies have analyzed the safety of high-dose dual therapy (HDDT) from a micro-ecological perspective.

View Article and Find Full Text PDF

Background: Circular (circ)RNAs have emerged as crucial contributors to cancer progression. Nonetheless, the expression regulation, biological functions, and underlying mechanisms of circRNAs in mediating hepatocellular carcinoma (HCC) progression remain insufficiently elucidated.

Methods: We identified circUCK2(2,3) through circRNA sequencing, RT-PCR, and Sanger sequencing.

View Article and Find Full Text PDF

Multiple myeloma (MM) remains an incurable hematological malignancy that necessitates the identification of novel therapeutic strategies. Here, we report that intracellular levels of very long chain fatty acids (VLCFAs) control the cytotoxicity of MM chemotherapeutic agents. Inhibition of VLCFA biosynthesis reduced cell death in MM cells caused by the proteasome inhibitor, bortezomib.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!