|
Atidarsagene Autotemcel
Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy treatment for metachromatic leukodystrophy (MLD) developed by Orchard Therapeutics. It contains an autologous CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector encoding the human arylsulfatase A Arylsulfatase A (or cerebroside-sulfatase) is an enzyme that breaks down sulfatides, namely cerebroside 3-sulfate into cerebroside and sulfate. In humans, arylsulfatase A is encoded by the ''ARSA'' gene. Pathology A deficiency is associated with m ... (''ARSA'') gene. Atidarsagene autotemcel was approved for medical use in the European Union in December 2020, Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. and in the United Kingdom in February 2021. Medical uses Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intravenous Infusion
Intravenous therapy (abbreviated as IV therapy) is a medical technique that administers fluids, medications and nutrients directly into a person's vein. The intravenous route of administration is commonly used for rehydration or to provide nutrients for those who cannot, or will not—due to reduced mental states or otherwise—consume food or water per os, by mouth. It may also be used to administer pharmaceutical drug, medications or other medical therapy such as blood transfusion, blood products or electrolytes to correct electrolyte imbalances. Attempts at providing intravenous therapy have been recorded as early as the 1400s, but the practice did not become widespread until the 1900s after the development of techniques for safe, effective use. The intravenous route is the fastest way to deliver medications and fluid replacement throughout the body as they are introduced directly into the circulatory system and thus quickly distributed. For this reason, the intravenous route o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Gene Therapy
Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.Gene Therapy Cli ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Metachromatic Leukodystrophy
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of sphingolipids. Leukodystrophies affect the growth and/or development of myelin, the fatty covering which acts as an insulator around nerve fibers throughout the central and peripheral nervous systems. MLD involves cerebroside sulfate accumulation. Metachromatic leukodystrophy, like most enzyme deficiencies, has an autosomal recessive inheritance pattern. Signs and symptoms Like many other genetic disorders that affect lipid metabolism, there are several forms of MLD, which are late infantile, juvenile, and adult. * In the ''late infantile form'', which is the most common form of MLD (50–60%), affected children begin having difficulty walking after the first year of life, usually at 15–24 months. Symptoms include muscle wasting and weakness, muscle rigidity, developmental delays, pr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orchard Therapeutics
An orchard is an intentional plantation of trees or shrubs that is maintained for food production. Orchards comprise fruit- or nut-producing trees which are generally grown for commercial production. Orchards are also sometimes a feature of large gardens, where they serve an aesthetic as well as a productive purpose. A fruit garden is generally synonymous with an orchard, although it is set on a smaller non-commercial scale and may emphasize berry shrubs in preference to fruit trees. Most temperate-zone orchards are laid out in a regular grid, with a grazed or mown grass or bare soil base that makes maintenance and fruit gathering easy. Most modern commercial orchards are planted for a single variety of fruit. While the importance of introducing biodiversity is recognized in forest plantations, it would seem to be beneficial to introduce some genetic diversity in orchard plantations as well by interspersing other trees through the orchard. Genetic diversity in an orchard would pr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Autologous
Autotransplantation is the transplantation of organs, tissues, or even particular proteins from one part of the body to another in the same person ('' auto-'' meaning "self" in Greek). The autologous tissue (also called autogenous, autogeneic, or autogenic tissue) transplanted by such a procedure is called an autograft or autotransplant. It is contrasted with allotransplantation (from other individual of the same species), syngeneic transplantation (grafts transplanted between two genetically identical individuals of the same species) and xenotransplantation (from other species). A common example is the removal of a piece of bone (usually from the hip) and its being ground into a paste for the reconstruction of another portion of bone. Autotransplantation, although most common with blood, bone, or skin, can be used for a wide variety of organs. One of the rare examples is autotransplantation of a kidney from one side of the body to the other. Kidney autotransplantation is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
CD34⁺
CD34 is a transmembrane phosphoglycoprotein protein encoded by the CD34 gene in humans, mice, rats and other species. CD34 derives its name from the cluster of differentiation protocol that identifies cell surface antigens. CD34 was first described on hematopoietic stem cells independently by Civin et al. and Tindle et al. as a cell surface glycoprotein and functions as a cell-cell adhesion factor. It may also mediate the attachment of hematopoietic stem cells to bone marrow extracellular matrix or directly to stromal cells. Clinically, it is associated with the selection and enrichment of hematopoietic stem cells for bone marrow transplants. Due to these historical and clinical associations, CD34 expression is almost ubiquitously related to hematopoietic cells; however, it is actually found on many other cell types as well. Function The CD34 protein is a member of a family of single-pass transmembrane sialomucin proteins that show expression on early haematopoietic and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hematopoietic Stem Cell
Hematopoietic stem cells (HSCs) are the stem cells that give rise to other blood cells. This process is called haematopoiesis. In vertebrates, the very first definitive HSCs arise from the ventral endothelial wall of the embryonic aorta within the (midgestational) aorta-gonad-mesonephros region, through a process known as endothelial-to-hematopoietic transition. In adults, haematopoiesis occurs in the red bone marrow, in the core of most bones. The red bone marrow is derived from the layer of the embryo called the mesoderm. Haematopoiesis is the process by which all mature blood cells are produced. It must balance enormous production needs (the average person produces more than 500 billion blood cells every day) with the need to regulate the number of each blood cell type in the circulation. In vertebrates, the vast majority of hematopoiesis occurs in the bone marrow and is derived from a limited number of hematopoietic stem cells that are multipotent and capable of extensive se ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lentiviral Vector
Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cells. This process can be performed inside a living organism (''in vivo'') or in cell culture (''in vitro''). Viruses have evolved specialized molecular mechanisms to efficiently transport their genomes inside the cells they infect. Delivery of genes or other genetic material by a vector is termed transduction and the infected cells are described as transduced. Molecular biologists first harnessed this machinery in the 1970s. Paul Berg used a modified SV40 virus containing DNA from the bacteriophage λ to infect monkey kidney cells maintained in culture. In addition to their use in molecular biology research, viral vectors are used for gene therapy and the development of vaccines. Key properties of a viral vector VIRAL VECTORS are tailored to their specific applications but generally share a few key properties. *''Safety'': Although viral vectors are occasionally created from pathogen ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Arylsulfatase A
Arylsulfatase A (or cerebroside-sulfatase) is an enzyme that breaks down sulfatides, namely cerebroside 3-sulfate into cerebroside and sulfate. In humans, arylsulfatase A is encoded by the ''ARSA'' gene. Pathology A deficiency is associated with metachromatic leukodystrophy, an autosomal recessive In genetics, dominance is the phenomenon of one variant (allele) of a gene on a chromosome masking or overriding the effect of a different variant of the same gene on the other copy of the chromosome. The first variant is termed dominant and t ... disease. Biochemistry Enzyme regulation Arylsulfatase A is inhibited by phosphate, which forms a covalent bond with the active site 3-oxoalanine. ; References Further reading * * * * * * * * * * * * * * * * * * External links GeneReviews/NCBI/NIH/UW entry on Arylsulfatase A Deficiency - Metachromatic Leukodystrophy OMIM entries on ARSA Deficiency * * * {{hydrolase-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
European Medicines Agency
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency (EMEA).Set up by EC Regulation No. 2309/93 as the European Agency for the Evaluation of Medicinal Products, and renamed by EC Regulation No. 726/2004 to the European Medicines Agency, it had the acronym EMEA until December 2009. The European Medicines Agency does not call itself EMA either – it has no official acronym but may reconsider if EMA becomes commonly accepted (secommunication on new visual identity an). The EMA was set up in 1995, with funding from the European Union and the pharmaceutical industry, as well as indirect subsidy from member states, its stated intention to harmonise (but not replace) the work of existing national medicine regulatory bodies. The hope was that this plan would not onl ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
NHS England
NHS England, officially the NHS Commissioning Board, is an executive non-departmental public body of the Department of Health and Social Care. It oversees the budget, planning, delivery and day-to-day operation of the commissioning side of the National Health Service in England as set out in the Health and Social Care Act 2012. It directly commissions NHS general practitioners, dentists, optometrists and some specialist services. The Secretary of State publishes annually a document known as the ''NHS mandate'' which specifies the objectives which the Board should seek to achieve. National Health Service (Mandate Requirements) Regulations are published each year to give legal force to the mandate. In 2018 it was announced that the organisation, while maintaining its statutory independence, would be merged with NHS Improvement, and seven "single integrated regional teams" would be jointly established. History NHS England is the operating name of the NHS Commissioning Board and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
_diagram_en.svg "Click for more on -> Hematopoietic Stem Cell")
