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Unlike pharmacological treatments which are administered locally or systemically as a series of interventions, gene therapy aims to establish sustained therapeutic effect after a single, local injection. [1] The main risk factors for osteoarthritis are age [2] [3] and body mass index, [4] [3] as such, OA is predominantly considered a disease of ...
Gene knockdown is an experimental technique by which the expression of one or more of an organism's genes is reduced. The reduction can occur either through genetic modification or by treatment with a reagent such as a short DNA or RNA oligonucleotide that has a sequence complementary to either gene or an mRNA transcript.
A disease-modifying osteoarthritis drug (DMOAD) is a disease-modifying drug that would inhibit or even reverse the progression of osteoarthritis. [1] Since the main hallmark of osteoarthritis is cartilage loss, a typical DMOAD would prevent the loss of cartilage and potentially regenerate it.
The disease causes motor neurons to degenerate, which eventually leads to neuron death and muscular degeneration. [66] Hundreds of mutations in the Cu/Zn superoxide dismutase (SOD1) gene have been found to cause ALS. [67] Gene silencing has been used to knock down the SOD1 mutant that is characteristic of ALS.
Ultimately, antiarthritic treatments aim to achieve disease remission or low disease activity if remission cannot be achieved and thereby improving quality of life. [ 8 ] The pharmacological effects of antiarthritic medications are typically exerted through the reduction of inflammation, suppression of the immune system and/or aid in easing pain.
DMARDs help control arthritis, but do not cure the disease. For that reason, if remission or optimal control is achieved with a DMARD, it is often continued as a maintenance dosage. Discontinuing a DMARD may reactivate disease or cause a "rebound flare", with no assurance that disease control will be re-established upon resumption of the ...
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