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Therapy: Gene Therapy

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Total 388 results found since Jan 2013.

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

Neural stem cell‐based treatment for neurodegenerative diseases
Human neurodegenrative diseases such as Parkinson's disease (PD), Huntington's disease (HD), amyotrophic lateral sclerosis (ALS) and Alzheimer's disease (AD) are caused by a loss of neurons and glia in the brain or spinal cord. Neurons and glial cells have successfully been generated from stem cells such as embryonic stem cells (ESCs), mesenchymal stem cells (MSCs) and neural stem cells (NSCs), and stem cell‐based cell therapies for neurodegenerative diseases have been developed. A recent advance in generatioin of a new class of pluripotent stem cells, induced pluripotent stem cells (iPSCs), derived from patients' own sk...
Source: Neuropathology - February 1, 2013 Category: Neurology Authors: Seung U. Kim, Hong J. Lee, Yun B. Kim Tags: Review Article Source Type: research

The Year in Review of Clinical Cardiac Electrophysiology
This past year saw multiple important advances in the field clinical cardiac electrophysiology. Seminal articles describing new anticoagulant drugs for stroke prevention in atrial fibrillation were published. New results that raise questions regarding the safety of dronedarone and several new promising techniques in AF ablation were described. Important articles that refine our understanding of the risk of sudden death among Wolff-Parkinson-White patients were published. In the basic and translational sciences, the application of gene therapy to the study and potential treatment of arrhythmias was described, whereas geneti...
Source: Journal of the American College of Cardiology: Cardiovascular Interventions - February 11, 2013 Category: Cardiology Source Type: research

Pain, pain, go away
(University of Texas Medical Branch at Galveston) University of Texas Medical Branch at Galveston researchers have been awarded a five-year, $1.8 million grant by the National Institute of Neurological Disorders and Stroke to apply the techniques of gene therapy to the problem of neuropathic pain -- that is, pain that arises from a malfunction in the nervous system.
Source: EurekAlert! - Medicine and Health - May 20, 2013 Category: Global & Universal Source Type: news

Comparison of vascular growth factors in the murine brain reveals placenta growth factor as prime candidate for CNS revascularization
Vascular bypass procedures in the central nervous system (CNS) remain technically challenging, hindered by complications and often failing to prevent adverse outcome such as stroke. Thus, there is an unmet clinical need for a safe and effective CNS revascularization. Vascular endothelial growth factors (VEGFs) are promising candidates for revascularization; however, their effects appear to be tissue-specific and their potential in the CNS has not been fully explored. To test growth factors for angiogenesis in the CNS, we characterized the effects of endothelium-specific growth factors on the brain vasculature and parenchym...
Source: Blood - August 1, 2013 Category: Hematology Authors: Gaal, E. I., Tammela, T., Anisimov, A., Marbacher, S., Honkanen, P., Zarkada, G., Leppanen, V.-M., Tatlisumak, T., Hernesniemi, J., Niemela, M., Alitalo, K. Tags: Vascular Biology, Gene Therapy Source Type: research

Lipid-Lowering Agents
The role of lipid lowering in reducing the risk of mortality and morbidity from cardiovascular disease (CVD) is well established. Treatment particularly aimed at decreasing low-density lipoprotein cholesterol (LDL-C) is effective in reducing the risk of death from coronary heart disease and stroke. Statins form the cornerstone of treatment. However, in some individuals with a high risk of CVD who are unable to achieve their target LDL-C due to either intolerance or lack of efficacy, there is the need for alternative therapies. This review provides an overview of the different classes of currently available lipid-lowering m...
Source: Journal of Cardiovascular Pharmacology and Therapeutics - August 12, 2013 Category: Cardiology Authors: Ewang-Emukowhate, M., Wierzbicki, A. S. Tags: Cardiovascular Pharmacology Core Review Source Type: research

Families hope 'Frankenstein science' lobby will not stop gene cure for mitochondrial disease
Change to IVF rules could make Britain the first country to allow therapy to change DNA in embryosDeniz Safak was five years old when he first displayed symptoms of the disease that would later take his life. "He started being sick and had intense, stroke-like seizures," his mother, Ruth, recalled.Doctors were baffled by the boy's condition and it took months before a diagnosis was made. Ruth and her husband, Erdhal, were told that Deniz was suffering from mitochondrial disease, an incurable condition that is passed from mother to child and can often be fatal.Deniz's condition continued to worsen. By the time he died last ...
Source: Guardian Unlimited Science - February 15, 2014 Category: Science Authors: Robin McKie Tags: Embryos IVF Genetics Children Biology News Health Medical research Society UK news The Observer Science Source Type: news

AAV-Mediated Gene Therapy for Atherosclerosis
Abstract The prognosis of patients with coronary artery disease and stroke has improved substantially over the last decade as a result of advances in primary and secondary preventive care as well as novel interventional approaches, including the development of drug-eluting stents and balloons. Despite this progress, however, cardiovascular disease remains the leading cause of death in industrialized nations. Sustained efforts to elucidate the underlying mechanisms of atherogenesis, reperfusion-induced cardiac injury, and ischemic heart failure have led to the identification of several target genes as key players ...
Source: Current Atherosclerosis Reports - August 3, 2014 Category: Cardiology Source Type: research

'Longevity Gene' One Key to Long Life, Research Suggests
As 'good' cholesterol goes up, odds of heart disease, stroke go down Source: HealthDay Related MedlinePlus Pages: Cholesterol, Genes and Gene Therapy, Seniors' Health
Source: MedlinePlus Health News - November 6, 2014 Category: Consumer Health News Source Type: news

The Use of Gene Therapy for Ablation of Atrial Fibrillation
In the United States, atrial fibrillation (AF) is the most common sustained cardiac arrhythmia affecting approximately six million patients and contributing to a greatly increased risk of stroke, heart failure (HF) and overall morbidity and mortality.1,2 The prevalence of AF is increasing as the average age of the population increases.3,4
Source: Radcliffe Cardiology - November 25, 2014 Category: Cardiology Authors: lravichander Source Type: research

ADAMTS13 and von Willebrand Factor in Thrombotic Thrombocytopenic Purpura.
Abstract Pathogenesis of thrombotic thrombocytopenic purpura (TTP) was a mystery for over half a century until the discovery of ADAMTS13. ADAMTS13 is primarily synthesized in the liver, and its main function is to cleave von Willebrand factor (VWF) anchored on the endothelial surface, in circulation, and at the sites of vascular injury. Deficiency of plasma ADAMTS13 activity (<10%) resulting from mutations of the ADAMTS13 gene or autoantibodies against ADAMTS13 causes hereditary or acquired (idiopathic) TTP. ADAMTS13 activity is usually normal or modestly reduced (>20%) in other forms of thrombotic microangi...
Source: Annual Review of Medicine - January 14, 2015 Category: Journals (General) Authors: Zheng XL Tags: Annu Rev Med Source Type: research

Targeted Gene Therapy for Ischemic Stroke
Exogenous delivery of angiogenic and neuroprotective genes has been shown to enhance innate compensatory responses after ischemic injury. However, there are certain barriers in translating gene-based therapy to the clinical setting. For example, systemic delivery of genes into the brain is prevented by the blood–brain barrier (BBB); intraventricular delivery results in nonspecific distribution and gene expression; and stereotactic injection of vectors into the ischemic penumbra requires an invasive procedure that can cause additional damage. This chapter describes an adeno-associated viral (AAV) vector with two prima...
Source: Springer protocols feed by Neuroscience - January 10, 2015 Category: Neuroscience Source Type: news

Changes in ventricular remodelling and clinical status during the year following a single administration of stromal cell-derived factor-1 non-viral gene therapy in chronic ischaemic heart failure patients: the STOP-HF randomized Phase II trial
Conclusions The blinded placebo-controlled STOP-HF trial demonstrated the safety of a single endocardial administration of pSDF-1 but failed to demonstrate its primary endpoint of improved composite score at 4 months after treatment. Through a pre-specified analysis the STOP-HF trial demonstrates the potential for attenuating LV remodelling and improving EF in high-risk ischaemic cardiomyopathy. The safety profile supports repeat dosing with pSDF-1 and the degree of left ventricular remodelling suggests the potential for improved outcomes in larger future trials.
Source: European Heart Journal - September 1, 2015 Category: Cardiology Authors: Chung, E. S., Miller, L., Patel, A. N., Anderson, R. D., Mendelsohn, F. O., Traverse, J., Silver, K. H., Shin, J., Ewald, G., Farr, M. J., Anwaruddin, S., Plat, F., Fisher, S. J., AuWerter, A. T., Pastore, J. M., Aras, R., Penn, M. S. Tags: Heart failure/cardiomyopathy Source Type: research

Batten disease may benefit from gene therapy
In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder.
Source: NINDS Press Releases and News: National Institute of Neurological Disorders and Stroke - November 11, 2015 Category: Neurology Source Type: news