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An American doctor flew to London this week to assess whether an 11-month old baby with a rare condition can be saved by an experimental medical treatment. Baby Charlie Gard has mitochondrial DNA depletion syndrome.
Gard has been at the center of a legal battle after his parents refused to turn off his life support when doctors said no more could be done for him.
Columbia University neuroscientist Michio Hirano says nucleoside bypass therapy has a 10 percent chance of improving Charlie’s condition.
Mitochondrial DNA depletion syndrome is caused by a gene mutation that prevents mitochondria from replacing damaged DNA. Nucleosides turn into nucleotides in the body, providing the missing components of DNA. Nucleoside bypass therapy provides the nucleosides needed to improve mitochondrial DNA.
The treatment has been tested on mice, but only 18 human patients have tried it. All of those patients had a mutation in a different gene than Charlie does, so no one knows for sure if the treatment will work.
Baby Charlie remains on life support as the legal wrangling continues.
RUNDOWN SHOWS:
1. Depiction of gene with mutation
2. Depiction of nucleosides and nucleotides; nucleotides complete DNA in mitochondria
3. Therapy tested on mice and 18 people
VOICEOVER (in English):
“Mitochondrial DNA depletion syndrome is caused by a gene mutation that prevents mitochondria from replacing damaged DNA.”
“Nucleosides turn into nucleotides in the body, providing the missing components of DNA.”
“Nucleoside bypass therapy provides the nucleosides needed to improve mitochondrial DNA.”
“The treatment has been tested on mice, but only 18 human patients have tried it.”
SOURCES: Stat, ABC News, Independent
https://www.statnews.com/2017/07/17/charlie-gard-treatment/
http://abcnews.go.com/Health/inside-experimental-treatment-doctors-charlie-gard/story?id=48723678
http://www.independent.co.uk/news/health/charlie-gard-us-citizenship-congress-american-treatment-uk-high-court-appeal-life-support-turn-off-a7848391.html
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