The spinal cord injury (SCI) is a medical and life-disrupting condition with devastating consequences for the physical, social, and professional welfare of patients, and there is no adequate treatment for it. At the same time, gene therapy has been studied as a promising approach for the treatment of neurological and neurodegenerative disorders by delivering remedial genes to the central nervous system (CNS), of which the spinal cord is a part. For gene therapy, multiple vectors have been introduced, including integrating lentiviral vectors and non-integrating adeno-associated virus (AAV) vectors. AAV vectors are a promising system for transgene delivery into the CNS due to their safety profile as well as long-term gene expression. Gene therapy mediated by AAV vectors shows potential for treating SCI by delivering certain genetic information to specific cell types. This review has focused on a potential treatment of SCI by gene therapy using AAV vectors.
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http://dx.doi.org/10.3390/biomedicines9060613 | DOI Listing |
Zhonghua Xue Ye Xue Za Zhi
December 2024
Department of Hematology, General Hospital, Tianjin Medical University, Tianjin Key Laboratory of Bone Marrow Failure and Malignant Hemopoietic Clone Control, Tianjin Institute of Hematology, Tianjin 300052, China.
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of hematopoietic stem cells induced by PIG-A gene mutations. It is clinically manifested by hemolysis, bone marrow failure, and high-risk concurrent thrombosis, which are life-threatening in severe cases. Significant progress has been made in the pathogenesis research and clinical diagnosis and treatment of PNH in recent years.
View Article and Find Full Text PDFZhonghua Xue Ye Xue Za Zhi
December 2024
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin 300020, China Tianjin Institutes of Health Science, Tianjin 301600, China.
This case report presents a patient with pediatric acute myeloid leukemia (AML) with RUNX1∷MTG16, admitted to the Blood Disease Hospital of the Chinese Academy of Medical Sciences in October 2023. He was 13 years old, with a chief complaint of fatigue for 20 days. Bone marrow smear revealed 17.
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December 2024
Laboratory of Precision Nanomedicine, The Shmunis School of Biomedicine and Cancer Research, George S. Wise Faculty of Life Sciences, Tel Aviv University, Tel-Aviv, Israel; Department of Materials Sciences and Engineering, Iby and Aladar Fleischman Faculty of Engineering, Tel Aviv University, Tel Aviv, Israel; Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel; Cancer Biology Research Center, Tel Aviv University, Tel Aviv, Israel. Electronic address:
Muscular dystrophies are a group of heterogenic disorders characterized by progressive muscle weakness, the most common of them being Duchenne muscular dystrophy (DMD). Muscular dystrophies are caused by mutations in over 50 distinct genes, and many of them are caused by different genetic mechanisms. Currently, none of these diseases have a cure.
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January 2025
Department of Radiation Oncology, Guangzhou Institute of Cancer Research, the Affiliated Cancer Hospital, Guangzhou Medical University, Guangzhou, China. Electronic address:
PANoptosis is a newly discovered complex programmed cell death (PCD) form. In the field of cancer research, PANoptosis is involved in multiple cell death pathways that affect tumor cell survival, proliferation, and response to treatment, serving as an innovative strategy for cancer therapy. Endocrine-disrupting chemicals (EDCs) impact the endocrine system, including cancer.
View Article and Find Full Text PDFInt Immunopharmacol
January 2025
Department of Urology, Shanghai Children's Medical Center, School of Medicine, Shanghai Jiao Tong University, No. 1678 Dongfang Road, Pudong New Area, Shanghai 200127, China. Electronic address:
Hedyotis diffusa Willd. (HDW), a traditional Chinese medicinal plant, exhibits a variety of pharmacological effects and has anticancer potential for a wide range of cancer types; Ferroptosis is a non-apoptosis-regulated cell death induced by iron accumulation and subsequent lipid peroxidation; and there is currently an increasing interest in the therapeutic role of ferroptosis in cancer. However, the effects of HDW on bladder cancer and its underlying molecular mechanisms remain largely unknown.
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