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Therapy: Gene Therapy

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Total 388 results found since Jan 2013.

Novartis ’ Spinal Drug Gets FDA Approval, $2 Million Price Tag
(CNN) — The US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants’ muscles to waste away, potentially killing them before age 2. And then came the price tag: $2.125 million for a one-time treatment. The gene therapy, called Zolgensma, will be marketed by AveXis, whose parent company is Novartis. “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” Dr. Ned Sharpless, the FDA’s acting commissioner, said in a statement Friday. ̶...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - May 24, 2019 Category: Consumer Health News Authors: Health – CBS Boston Tags: Health News CNN Novartis Source Type: news

Recent advances in the therapeutic uses of chondroitinase ABC.
Abstract Many studies, using pre-clinical models of SCI, have demonstrated the efficacy of chondroitinase ABC as a treatment for spinal cord injury and this has been confirmed in laboratories worldwide and in several animal models. The aim of this review is report the current state of research in the field and to compare the relative efficacies of these new interventions to improve outcomes in both acute and chronic models of SCI. We also report new methods of chondroitinase delivery and the outcomes of two clinical trials using the enzyme to treat spinal cord injury in dogs and disc herniation in human patients. ...
Source: Experimental Neurology - August 5, 2019 Category: Neurology Authors: Muir E, De Winter F, Verhaagen J, Fawcett J Tags: Exp Neurol Source Type: research

Curing hemoglobinopathies: challenges and advances of conventional and new gene therapy approaches.
Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD) are the most common monogenic diseases worldwide, with a global carrier frequency of over 5%. With migration they are becoming more common worldwide, making their management and care an increasing concern for health care systems.BT is characterized by an imbalance in the α/β-globin chain ratio, ineffective erythropoiesis, chronic hemolytic anemia, and compensatory haemopoietic expansion. Globally, there are over 25,000 births each year with transfusion-dependent thalassemia (TDT). The current available treatment f...
Source: Mediterranean Journal of Hematology and Infectious Diseases - October 30, 2019 Category: Hematology Source Type: research

Sickle cell disease as a vascular disorder.
Authors: Ofori-Acquah SF Abstract Introduction: In sickle cell disease (SCD), hemoglobin S (HbS) red blood cells (RBCs) are characteristically deformed and inflexible. Often breaking down in the circulation, they exhibit increased adhesive properties with the endothelium and activated neutrophils and platelets, increasing the risk of occlusion of the microcirculation. SCD is categorized into two sub-phenotypes: hyperhemolytic, associated with priapism, leg ulcers, pulmonary hypertension, and stroke, and high hemoglobin/viscosity, which may promote vaso-occlusion-associated pain, acute chest syndrome, and osteonecro...
Source: Expert Review of Hematology - May 6, 2020 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Attenuation of Oxidative Injury with Targeted Expression of NOX2 shRNA Prevents Onset and Maintenance of Electrical Remodeling in the Canine Atrium: A Novel Gene Therapy Approach to Atrial Fibrillation.
Conclusions: NOX2 oxidative injury: a) underlies onset as well as maintenance of electrical remodeling in AF, and b) can be successfully prevented with a novel, gene-based approach. Future optimization of this approach may lead to a novel, mechanism-guided therapy for AF. PMID: 32686471 [PubMed - as supplied by publisher]
Source: Circulation - July 19, 2020 Category: Cardiology Authors: Yoo S, Pfenniger A, Hoffman J, Zhang W, Ng J, Burrell A, Johnson DA, Gussak G, Waugh T, Bull S, Benefield B, Knight BP, Passman R, Wasserstrom JA, Aistrup GL, Arora R Tags: Circulation Source Type: research

Granulocyte-colony stimulating factor gene therapy as a novel therapeutics for stroke in a mouse model
Global ischemia is the resulting effect of a cardiopulmonary arrest (CPA). Presently there is no effective treatment to address neurological deficits in patients who survived a CPA. Granulocyte-colony stimulat...
Source: Journal of Biomedical Science - October 30, 2020 Category: Biomedical Science Authors: Janet M. Menzie-Suderam, Jigar Modi, Hongyaun Xu, Andrew Bent, Paula Trujillo, Kristen Medley, Eugenia Jimenez, Jessica Shen, Michael Marshall, Rui Tao, Howard Prentice and Jang-Yen Wu Tags: Research Source Type: research

Long noncoding RNA MEG3 contributes to dysfunction of brain microvascular endothelial cells after intracerebral hemorrhage by regulating the miR-1930-5p/Mllt1 axis.
CONCLUSIONS: In conclusion, lncRNA MEG3 promoted the dysfunction of BMECs by modulating the miR-1930-5p/Mllt1 axis, which provides a potential target in gene therapy for brain injury following ICH. PMID: 33127454 [PubMed - as supplied by publisher]
Source: Brain Research Bulletin - October 27, 2020 Category: Neurology Authors: Li Z, Han L, Liang Q, Huang Z Tags: Brain Res Bull Source Type: research

Therapeutic advances in Fabry disease: The future awaits.
Abstract Fabry disease (FD) is an X-linked disorder caused by mutations in GLA gene responsible for coding of the lysosomal enzyme alpha-galactosidase A(α-GAL). The resultant accumulation of globotriaosylceramide (Gb-3) leads to multisystemic disease including progressive chronic kidney disease, hypertrophic cardiomyopathy, stroke, angiokeratomas and corneal whorls. Current treatments include enzyme replacement therapy (ERT), along with recent advent of chaperone therapy. ERT has not shown to have dramatic improvement in outcomes for all organ systems, with benefit mostly seen in kidney disease and reduction in l...
Source: Biomedicine and pharmacotherapy = Biomedecine and pharmacotherapie - November 1, 2020 Category: Drugs & Pharmacology Authors: Kant S, Atta MG Tags: Biomed Pharmacother Source Type: research

Recent Advances in Gene Therapy for Atrial Fibrillation
This article is protected by copyright. All rights reserved.
Source: Journal of Cardiovascular Electrophysiology - May 30, 2021 Category: Cardiology Authors: Shin Yoo, Gail Elizabeth Geist, Anna Pfenniger, Markus Rottmann, Rishi Arora Tags: REVIEW Source Type: research

New Directions in Therapeutic Angiogenesis and Arteriogenesis in Peripheral Arterial Disease
Circ Res. 2021 Jun 11;128(12):1944-1957. doi: 10.1161/CIRCRESAHA.121.318266. Epub 2021 Jun 10.ABSTRACTThe prevalence of peripheral arterial disease (PAD) in the United States exceeds 10 million people, and PAD is a significant cause of morbidity and mortality across the globe. PAD is typically caused by atherosclerotic obstructions in the large arteries to the leg(s). The most common clinical consequences of PAD include pain on walking (claudication), impaired functional capacity, pain at rest, and loss of tissue integrity in the distal limbs that may lead to lower extremity amputation. Patients with PAD also have higher t...
Source: Circulation Research - June 10, 2021 Category: Cardiology Authors: Brian H Annex John P Cooke Source Type: research

Mitochondrial Disorders
CONCLUSION: Patients with mitochondrial diseases have highly varied manifestations and can thus present to physicians in practically any branch of medicine. A correct diagnosis is the prerequisite for genetic counseling and for the initiation of personalized treatment.PMID:34158150 | DOI:10.3238/arztebl.m2021.0251
Source: Deutsches Arzteblatt International - June 23, 2021 Category: General Medicine Authors: Thomas Klopstock Claudia Priglinger Ali Yilmaz Cornelia Kornblum Felix Distelmaier Holger Prokisch Source Type: research