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1:26
Gene Therapy Explained: Can We Really Fix Genetic Diseases?
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Spinal muscular atrophy type 2 is a genetic disease where motor neurons slowly fail. Kids are usually diagnosed in early childhood. They can sit but often cannot walk. Intelligence is normal. The problem is muscle weakness not the brain. Early diagnosis and modern therapies are changing outcomes but it is still a lifelong neuromuscular disease. #genetic #medical #science #stem #interesting
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As more high-cost genetic therapies move toward approval, employers may have to confront difficult decisions, according to economist Jonathan Gruber: cover a potentially lifesaving treatment that could bankrupt them or deny and leave workers without options. Gruber likens it to a coming tsunami that’s going to overwhelm the employer-sponsored insurance system. “What happens when you have genetic cell and gene therapies that treat cancer or heart disease, which are much more common? That's when t
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Meet Baislee, our resilient daughter navigating a rare genetic disorder. This video offers a snapshot of her diagnoses, therapies, and the amazing specialists guiding her path. #raregeneticdisorder #raregeneticcondition #specialneedsfamily #specialneedsmom #feedingtherapy #latinafollowtrain #BaisleeJourney
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Sanfilippo Syndrome is a degenerative terminal genetic disorder, often referred to as childhood Alzheimer’s. Life expectancy is mid to late teens, with no current FDA approved treatment, clinical trials/experimental therapies only. #sanfilipposyndrome #fyp #savingliv #genetics
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“It’s Just ‘Junk DNA’” #wait. Sources- PMID 42726509 PMID 26015273 Microproteins, short chains of amino acids once dismissed as useless genetic noise, are emerging as a promising frontier in precision oncology. Modern sequencing technologies have revealed that tiny regions of DNA previously labeled as non-coding actually produce these functional, highly specialized proteins. Because many microproteins are expressed exclusively inside cancer cells or regulate critical metabolic pathways that fuel
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#greenscreen Thanks to CRISPR, genes can be editied to fix genetic conditions like epidermolysis bullosa but what else may this be used for #crispr #geneediting #epidermolysisbullosa #blisters #skin #dermatologist
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