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Therapy: Gene Therapy
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Total 388 results found since Jan 2013.
Fabry disease - current data and therapeutic approaches
Rom J Morphol Embryol. 2021 Jan-Mar;62(1):5-11. doi: 10.47162/RJME.62.1.01.ABSTRACTFabry disease represents an X-linked inherited disorder resulting in the accumulation of globotriaosylceramide (Gb3). This review explains the clinical manifestations and the possible therapies for this condition. Fabry disease is considered the second most frequent lysosomal storage disease. More than 1000 mutations of the galactosidase alpha (GLA) gene associated with this disorder have been identified. Pain, either episodic crises or chronic pain, is one of the earliest symptoms in Fabry disease. Gastrointestinal, ocular, ear or skeletal ...
Source: Romanian Journal of Morphology and Embryology - October 5, 2021 Category: General Medicine Authors: Ilie Robert Dinu Ştefan George Firu Source Type: research
Mesenchymal Stromal Cells (MSCs): a promising tool for cell-based angiogenic therapy
Curr Gene Ther. 2021 Sep 16. doi: 10.2174/1566523221666210917114353. Online ahead of print.ABSTRACTThe Mesenchymal stromal cells (MSCs) are a diverse subset of adult multipotent precursors, known for their potential therapeutic properties in regenerative medicine mainly sustained by paracrine effects, through secretion of a variety of biologically active molecules. MSC secretome includes a wide range of soluble protein factors, composed of growth factors and cytokines, and vesicular components, which transfer proteins and genetic material modulating the host microenvironment. In particular, MSC-derived secretome mediates t...
Source: Current Gene Therapy - September 17, 2021 Category: Genetics & Stem Cells Authors: Cristiana Ulpiano Cl áudia L da Silva Gabriel A Monteiro Source Type: research
NIH-funded study finds gene therapy may restore missing enzyme in rare disease
(NIH/National Institute of Neurological Disorders and Stroke) A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare neurological disorder that develops in infancy and leads to near absent levels of certain brain chemicals, serotonin and dopamine, that are critical for movement, behavior, and sleep.
Source: EurekAlert! - Medicine and Health - July 16, 2021 Category: International Medicine & Public Health Source Type: news
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
