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Mickey Tsai

Parkinson's disease sufferer Sheila Roy can write for the first time in 15 years thanks... - 0 views

www.dailymail.co.uk/...s-thanks-new-gene-therapy.html

gene therapy disease

shared by Mickey Tsai on 15 Apr 12 - No Cached
  • one of only 15 people worldwide to undergo the radical treatment, which involves inserting corrective genes into the brain
  • The genes provide the coded instructions for proteins needed to make dopamine, a brain chemical essential for proper control of movement.
  • Lack of dopamine leads to the symptoms of tremor, stiffness and poor balance associated with Parkinson’s.
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  • Mrs Roy is taking part in an early-stage study of the ProSavin therapy
  • Following her treatment Sheila Roy has been able to carry out tasks like writing, something she has been unable to do for 15 years
  • Gene therapies hold great promise for people with Parkinson’s in the future, as they could mean an end to the daily regime of drugs that most people with the condition currently face.
  • Mickey Tsai on 15 Apr 12
     
    A woman with Parkinson's was able to write for the first time in 15 years because of gene therapy. Parkinson's includes symptoms such as tremors, loss of balance which makes it impossible to do even simple tasks. Doctors injected a modified virus carrying the genes to the motor centre of her brain which provide coded instructions for proteins needed to make dopamine. Lack of dopamine leads of the symptoms associated with Parkinson's. This is part of the ProSavin therapy developed by Oxford BioMedica. Gene therapies hold a lot of hope for people with Parkinson's that could end the daily routine of drugs that most of them go through.
Pop karnchanapimonkul

Sight Seen: Gene Therapy Restores Vision in Both Eyes: Scientific American - 0 views

www.scientificamerican.com/article.cfm

gene therapy vision disease enzyme

shared by Pop karnchanapimonkul on 13 Apr 12 - No Cached
  • gene therapy to treat blindness in 12 adults and children with Leber's congenital amaurosis (LCA), a rare inherited eye disease that destroys vision by killing photoreceptors—light-sensitive cells in the retina at the back of the eye.
  • genetic mutations in retinal cells. One mutated gene that causes the disorder is named RPE65. An enzyme encoded by RPE65 helps break down a derivative of vitamin A called retinol into a substance that photoreceptors need to detect light and send signals to the brain.
  • injected a harmless virus carrying normal copies of RPE65
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  • subsequently began producing the enzyme
  • proved so much they no longer met the criteria for legal blindness
  • injected the functional genes into the previously untreated eye
  • improved as soon as two weeks after the operation: They could navigate an obstacle course, even in dim light, avoiding objects that had tripped them up before, as well as recognize people's faces and read large signs
  • brains were much more responsive to optical input as well.
  • second round of gene therapy further strengthened the brain's response to the initially treated eye as well as the newly treated one
  • that neuroplasticity plays a role
  • visual cortex responding to the newly flowing channel of information from the second eye bolster activity in areas of the visual cortex responding to the initially treated eye.
  • Pop karnchanapimonkul on 13 Apr 12
     
    Article about how the enzyme produced from gene therapy is used to cure blindness in an eye genetic disease.
chanon chiarnpattanodom

Stem cell therapy could repair some heart damage - Yahoo! News - 2 views

news.yahoo.com/HQDc2VjdGlvbnMEdGVzdAM-;_ylv=3

shared by chanon chiarnpattanodom on 02 Apr 12 - No Cached
  • Patients with advanced heart disease who received an experimental stem cell therapy
  • Study authors described the trial as the largest to date to examine stem cell therapy as a route to repairing the heart in patients with chronic ischemic heart disease and left ventricular dysfunction.
  • injections of their own stem cells, taken from their bone marrow, into damaged areas of their hearts.
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  • The patients -- 82 of whom were men -- all had chronic heart disease, along with either heart failure or angina or both, and their left ventricles were pumping at less than 45 percent of capacity.
  • None of the participant
  • eligible for revascularization surgery
  • heart disease was so advanced
  • Those who received the stem cell therapy saw a small but significant boost in the heart's ability to pump blood, measuring the increase from the heart's main pumping chamber at 2.7 percent more than placebo patients.
  • However, other factors showed no improvement
  • heart's maximum oxygen consumption did not change
  • defects in the heart were not healed by the treatment
  • This is the kind of information we need in order to move forward with the clinical use of stem cell therapy," said lead investigator Emerson Perin
  • "With this mapping procedure, we have a roadmap to the heart muscle," said Perin
  • Heart disease is the leading killer in the United States, claiming nearly 600,000 lives per year, according to the US Centers for Disease Control and Prevention.
  • chanon chiarnpattanodom on 02 Apr 12
     
    A recent experiment was done on elderly patients who had heart diseases, those that had progressed too far for coronary surgery. Patients were injected with their own stem cells in the bone marrow into areas in the heart. The pumping capacity did improve a little, but overall the oxygen use and the defects did not change. This is a stepping stone towards using stem cells to treat people in difficult situations where a normal surgery would not.
orasa sukmark

Gene therapy - 0 views

www.sciencedaily.com/...gene_therapy.htm

gene therapy

shared by orasa sukmark on 02 Apr 12 - No Cached
  • orasa sukmark on 02 Apr 12
     
    general info on gene therapy
avikan

New Hope Of a Cure For H.I.V. - NYTimes.com - 0 views

query.nytimes.com/...fullpage.html

shared by avikan on 11 Apr 12 - No Cached
  • So people with H.I.V. now must take drugs every day for life, which some researchers say is not a sustainable solution for tens of millions of infected people.
  • CCR5,
  • This is what was done with the Trenton patient. Some of the man's white blood cells were removed from his body and treated with a gene therapy developed by Sangamo BioSciences. The therapy induced the cells to produce proteins called zinc-finger nucleases that can disrupt the CCR5 gene.
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    • avikan
      avikan on 11 Apr 12
       
      Millions of people worldwide are currently affected by HIV and many have died from AIDS. Scientists have been trying for many years to find a cure for the epidemic, but now many are trying to find a way to prevent the passing on of the virus for future generations. Although no definite treatment has been discovered yet, recent findings have shown promising results for the future. 
    • avikan
      avikan on 11 Apr 12
       
      With the fast developing biological technologies we are seeing today, scientists hope's are growing stronger.  Maybe one of us one day will be a part of the phenomenon, in search of a way to help the millions affected by the epidemic  
    • avikan
      avikan on 11 Apr 12
       
      CD4 cells initiate the body's response to infections.
    • avikan
      avikan on 11 Apr 12
       
      Many forms of HIV, initially use CCR5 to enter and infect host cells. A few individuals carry a mutation known as CCR5 delta 32 in the CCR5 gene, protecting them against these strains of HIV.
    • avikan
      avikan on 11 Apr 12
       
      For over 30 years scientists have been trying to find a cure for the HIV/AIDs epidemic and so far have been unsuccessful 
    • avikan
      avikan on 11 Apr 12
       
      Incase some of us forgot, AIDs stands for Acquired immune deficiency syndrome, a disease in which there is a severe loss of the body's cellular immunity. And HIV stands for Human immunodeficiency virus, a virus that causes AIDs
Nickyz P.

GEN | Magazine Articles: Firm Focuses Operations on Gene Silencing - 0 views

www.genengnews.com/...4004

shared by Nickyz P. on 16 Apr 12 - No Cached
  • It is developing therapeutics to prove the validity of ddRNAi in treating cancer, infectious diseases, and disorders of the central nervous system.
  • The ddRNAi platform focuses on the long-term downregulation of genes, making it suitable for targeting chronic life-threatening diseases. “We are silencing genes instead of introducing new genes, which separates us from traditional gene therapy companies,” Dr. French asserts.
  • “This targeted treatment markedly enhanced the benefits of radiation therapy in both cellular and tumor models,” the researchers concluded. Other radiotherapy-resistant tumors may benefit from the shRNAs created for the prostate cancer study.
Nitchakan Chaiprukmalakan

How a single gene mutation leads to uncontrolled obesity - 0 views

www.sciencedaily.com/...120318143904.htm

shared by Nitchakan Chaiprukmalakan on 13 Apr 12 - No Cached
  • Researchers at Georgetown University Medical Center have revealed how a mutation in a single gene is responsible for the inability of neurons to effectively pass along appetite suppressing signals from the body to the right place in the brain.
  • The research team specifically found that a mutation in the brain-derived neurotrophic factor (Bdnf) gene in mice does not allow brain neurons to effectively pass leptin and insulin chemical signals through the brain. In humans, these hormones, which are released in the body after a person eats, are designed to "tell" the body to stop eating. But if the signals fail to reach correct locations in the hypothalamus, the area in the brain that signals satiety, eating continues.
  • He has found that the gene produces a growth factor that controls communication between neurons.
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  • The Bdnf gene generates one short transcript and one long transcript. He discovered that when the long-form Bdnf transcript is absent, the growth factor BDNF is only synthesized in the cell body of a neuron but not in its dendrites. The neuron then produces too many immature synapses, resulting in deficits in learning and memory in mice. Xu also found that the mice with the same Bdnf mutation grew to be severely obese
  • large-scale genome-wide association studies showed Bdnf gene variants are, in fact, linked to obesity.
  • both leptin and insulin stimulate synthesis of BDNF in neuronal dendrites in order to move their chemical message from one neuron to another through synapses. The intent is to keep the leptin and insulin chemical signals moving along the neuronal highway to the correct brain locations, where the hormones will turn on a program that suppresses appetite.
  • "If there is a problem with the Bdnf gene, neurons can't talk to each other, and the leptin and insulin signals are ineffective, and appetite is not modified
  • One possible strategy would be to produce additional long-form Bdnf transcript using adeno-associated virus-based gene therapy, Xu says. But although this kind of gene therapy has proven to be safe, it is difficult to deliver across the brain blood barrier,
adisa narula

Breaking the Silence: The Rise of Epigenetic Therapy - 0 views

jnci.oxfordjournals.org/...874.full

cancer DNA genetics research epigenetic

shared by adisa narula on 12 Apr 12 - No Cached
  • adisa narula on 12 Apr 12
     
    Cancer epigenetics is hot. At the annual meeting of the American Association for Cancer Research in April, once-obscure principal investigators were feted by gaggles of admirers and many poster presenters mobbed by the curious. "It's one of the hottest areas of basic biology," said Paul Workman, Ph.D., director of cancer therapeutics at Cancer Research U.K.
Kantham Hongdusit

Gene transfer to human joints: Progress toward a gene therapy of arthritis - 0 views

www.pnas.org/...8698.full

shared by Kantham Hongdusit on 16 Apr 12 - No Cached
  • Kantham Hongdusit on 16 Apr 12
     
    Rheumatoid Arthritis is an incurable disease that is often difficult to treat. The article describes how a transfer of cultured synovial fibroblasts can increase the amount of IL-1Ra receptor, which offers a therapeutic gene that can be used to obtain intra articular transgene expression.
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