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Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia.
JCI Insight
May 2024
Division of Hematopoietic Innovative Therapies, CIEMAT, Madrid, Spain.
Article Synopsis
- This study introduces a promising approach for gene therapy in Diamond Blackfan anemia (DBA), focusing on patients with mutations in the RPS19 gene and demonstrating a safe and effective treatment method.
- Unlike Fanconi anemia, DBA patients have a healthy reserve of hematopoietic stem cells, making them suitable candidates for this therapy without significant complications.
- Two novel lentiviral vectors were developed to deliver RPS19, showing successful restoration of red blood cell development in lab experiments, along with confirming safety and potential long-term benefits in hematopoietic cell function.
Correction: Lentivirus-Mediated Overexpression of SIVA-1 Reverses Cisplatin Resistance in Gastric Cancer in vitro.
Cell Biochem Biophys
March 2024
Department of Gastrointestinal and Peripheral Vascular Surgery, People's Hospital of Guangxi Zhuang Autonomous Region, Nanning, China.
Lentiviral Mediated Gene Transfer Corrects the Defects Associated With Deficiency of Adenosine Deaminase Type 2.
Front Immunol
May 2022
Infection, Immunity, Inflammation Department, University College London (UCL) Great Ormond Street Institute of Child Health, London, United Kingdom.
Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease caused by bi-allelic loss-of-function mutations in . Treatment with anti-TNF is effective for the autoinflammatory and vasculitic components of the disease but does not correct marrow failure or immunodeficiency; and anti-drug antibodies cause loss of efficacy over time. Allogeneic haematopoietic stem cell transplantation may be curative, but graft versus host disease remains a significant concern.
View Article and Find Full Text PDF[Effects and mechanism of hepatocyte growth factor-modified human adipose mesenchymal stem cells on wound healing of full-thickness skin defects in diabetic rats].
Zhonghua Shao Shang Za Zhi
September 2021
Department of Burns and Cutaneous Surgery, Burn Center of PLA, the First Affiliated Hospital of Air Force Medical University, Xi'an 710032, China.
To investigate the effects and mechanism of hepatocyte growth factor (HGF)-modified human adipose mesenchymal stem cells (ADSCs) on the wound healing of full-thickness skin defects in diabetic rats. The experimental research method was adopted. The discarded abdominal adipose tissue was collected from a 35-year-old healthy female who underwent abdominal liposuction in the Department of Plastic Surgery of the First Affiliated Hospital of Air Force Medical University in December 2019.
View Article and Find Full Text PDFAutophagy is required during high MUC2 mucin biosynthesis in colonic goblet cells to contend metabolic stress.
Am J Physiol Gastrointest Liver Physiol
November 2021
Department of Microbiology, Immunology and Infectious Diseases, University of Calgary Health Sciences Centre, Calgary, Alberta, Canada.
Goblet cells are specialized for the production and secretion of MUC2 glycoproteins that forms a thick layer covering the mucosal epithelium as a protective barrier against noxious substances and invading microbes. High MUC2 mucin biosynthesis induces endoplasmic reticulum (ER) stress and apoptosis in goblet cells during inflammatory and infectious diseases. Autophagy is an intracellular degradation process required for maintenance of intestinal homeostasis.
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