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Therapy: Gene Therapy
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Total 388 results found since Jan 2013.
Gene therapy of adeno-associated virus (AAV) vectors in preclinical models of ischemic stroke
CNS Neurosci Ther. 2023 Aug 8. doi: 10.1111/cns.14392. Online ahead of print.ABSTRACTStroke has been associated with devastating clinical outcomes, with current treatment strategies proving largely ineffective. Therefore, there is a need to explore alternative treatment options for addressing post-stroke functional deficits. Gene therapy utilizing adeno-associated viruses (AAVs) as a critical gene vector delivering genes to the central nervous system (CNS) gene delivery has emerged as a promising approach for treating various CNS diseases. This review aims to provide an overview of the biological characteristics of AAV vec...
Source: CNS Neuroscience and Therapeutics - August 8, 2023 Category: Neuroscience Authors: Jing Wang Mengna Zhu Jingyi Sun Lina Feng Mingfeng Yang Baoliang Sun Leilei Mao Source Type: research
Sestrin2 as a Potential Target in Hypertension
Diagnostics (Basel). 2023 Jul 14;13(14):2374. doi: 10.3390/diagnostics13142374.ABSTRACTHypertension is a highly complex, intricate condition affecting millions of individuals across the globe. Nearly half of adults in the United States are diagnosed with hypertension, with incident rates projected to rise over the next decade. Hypertension is a precursor to many cardiovascular diseases including atherosclerosis, stroke, myocardial infarction, heart failure, and peripheral artery disease. This review describes the major processes contributing to the development of hypertension and how Sestrin2 (Sesn2), an antioxidative prot...
Source: Atherosclerosis - July 29, 2023 Category: Cardiology Authors: Steven Didik Hao Wang Adewale Segun James Lily Slotabec Ji Li Source Type: research
Deficiency of adenosine deaminase 2 (DADA2): Review
Best Pract Res Clin Rheumatol. 2023 Jun 15:101844. doi: 10.1016/j.berh.2023.101844. Online ahead of print.ABSTRACTThe deficiency of adenosine deaminase 2 (DADA2) is an autosomal recessive disease caused by loss-of-function (LOF) mutations in the ADA2 gene and was first described in 2014. Initially, it was described as vasculopathy/vasculitis that mostly affected infants and young children and closely resembled polyarteritis nodosa (PAN). Skin rash and ischemic/hemorrhagic stroke are predominant symptoms. However, the clinical spectrum of DADA2 has continued to expand since then. It has now been reported in adults as well. ...
Source: Cell Research - June 16, 2023 Category: Cytology Authors: Vikas Sharma Prateek Deo Aman Sharma Source Type: research
Bs-452758-4 combined gene therapy approach targets parasympathetic signaling and oxidative stress to attenuate persistent atrial fibrillation development in a canine model
Atrial fibrillation (AF) is the most common arrhythmia, a major risk factor for stroke and is associated with substantial morbidity and mortality. Unfortunately, current pharmacological and interventional treatment approaches are suboptimal. Parasympathetic nervous system (PNS) activity upregulation and oxidative stress (OS), mainly generated by NADPH oxidase 2 (NOX2), have been shown as key players in AF induction and maintenance. A deeper understanding of these molecular mechanisms underlying AF and their interplay will be instrumental to the development of new, mechanism-targeted therapies for AF.
Source: Heart Rhythm - May 1, 2023 Category: Cardiology Authors: Aleksei Mikhailov, Azaria Panni, Anna Pfenniger, Shin Yoo, David Johnson, Wenwei Zhang, Markus Rottmann, William Marszalec, Asish Ghosh, Rod S. Passman, Bradley P. Knight, Rishi Arora Source Type: research
Current and Novel Therapeutical Approaches of Classical Homocystinuria in Childhood With Special Focus on Enzyme Replacement Therapy, Liver-Directed Therapy and Gene Therapy
J Clin Med Res. 2023 Feb;15(2):76-83. doi: 10.14740/jocmr4843. Epub 2023 Feb 28.ABSTRACTClassical homocystinuria is a hereditary defect of the enzyme cystathionine beta synthase, which is produced in the liver. If this enzyme fails, the synthesis pathway of cysteine from methionine is interrupted, leading to the accumulation of homocysteine in the blood plasma and homocysteine in the urine. After birth, the children are unremarkable except for the characteristic laboratory findings. Symptoms rarely appear before the second year of life. The most common symptom is a prolapse of the crystalline lens. This finding is seen in ...
Source: Clin Med Res - March 10, 2023 Category: Research Authors: Stefan Bittmann Gloria Villalon Elena Moschuring-Alieva Elisabeth Luchter Lara Bittmann Source Type: research
Organ function indications and potential improvements following curative therapy for sickle cell disease
Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):277-282. doi: 10.1182/hematology.2022000372.ABSTRACTCurative therapies for sickle cell disease include allogeneic hematopoietic stem cell transplantation (HSCT) and gene-modified autologous stem cell transplantation. HSCT has been used for 30 years with success measured by engraftment, symptom control, graft-vs-host disease (GVHD) risk, organ toxicity, and immune reconstitution. While human leukocyte antigen-matched sibling donor (MSD) transplants have excellent outcomes, alternate donor transplants (unrelated/haploidentical) are just beginning to overcome GVHD and...
Source: Hematology ASH Education Program - December 9, 2022 Category: Hematology Authors: Monica L Hulbert Allison A King Shalini Shenoy Source Type: research
