PITTSBURGH (KDKA) — Doctors in Britain say a radical new treatment for Parkinson’s disease could improve the lives of millions who suffer from the disease.
Sheila Roy says she can finally enjoy life at her countryside home in England.
She’s lived with Parkinson’s disease for 17 years, but a team of Oxford scientists developed a treatment that changed her life.
“I can see a glimmer of the person I used to be now, which is really exciting,” said Roy.
She is one of only 15 people in the world to take part in a gene therapy experiment. ....
Parkinson's disease (also known as Parkinson disease, Parkinson's, idiopathic parkinsonism, primary parkinsonism, PD, or paralysis agitans) is a degenerative disorder of the central nervous system. The motor symptoms of Parkinson's disease result from the death of dopamine-generating cells in the substantia nigra, a region of the midbrain; the cause of this cell death is unknown. Early in the course of the disease, the most obvious symptoms are movement-related; these include shaking, rigidity, slowness of movement and difficulty with walking and gait. Later, cognitive and behavioural problems may arise, with dementia commonly occurring in the advanced stages of the disease. Other symptoms include sensory, sleep and emotional problems. PD is more common in the elderly, with most cases occurring after the age of 50.
Originally posted by xuenchen
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.
Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID. Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.
Although early clinical failures led many to dismiss gene therapy as over-hyped, clinical successes in 2009-2011 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber's Congenital Amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, and Parkinson's disease. These recent clinical successes have led to a renewed interest in gene therapy, with several articles in scientific and popular publications calling for continued investment in the field.
Originally posted by autowrench
reply to post by xuenchen
This is great. May be some hope for Micheal J. Fox, one of my favorite actors. Loved him in "Hollywood Doc".