Building Gene Therapies That Address Gain And Loss Of Function Simultaneously RARECast podcast

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Building Gene Therapies that Address Gain and Loss of Function Simultaneously

2M ago 40:08

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For some genetic diseases, there is not only the need to replace the function of a gene that is lost, but to also address toxicities that a mutated gene may cause. There is currently no available treatment targeting diseases that result from both loss and gain of function mutations. NGGT uses dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function. We spoke to Guang Qu, chief operating officer and co-founder of NGGT, about the company’s approach to gene therapies, how it is leveraging its platform technologies to cost-effectively accelerate development of these therapies, and the pipeline it is building.

… continue reading

520 episoder

#Health Industry #Business #Health Care #Rare Disease #RARECast #Health and Well-Being

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Building Gene Therapies that Address Gain and Loss of Function Simultaneously

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published 2M ago

Dela

MP3•Episod hem

Innehåll tillhandahållet av RARECast. Allt poddinnehåll inklusive avsnitt, grafik och podcastbeskrivningar laddas upp och tillhandahålls direkt av RARECast eller deras podcastplattformspartner. Om du tror att någon använder ditt upphovsrättsskyddade verk utan din tillåtelse kan du följa processen som beskrivs här https://sv.player.fm/legal.

For some genetic diseases, there is not only the need to replace the function of a gene that is lost, but to also address toxicities that a mutated gene may cause. There is currently no available treatment targeting diseases that result from both loss and gain of function mutations. NGGT uses dual-functional vectors to simultaneously remove harmful, mutated genes and replace them with normal, healthy genes to restore cellular function. We spoke to Guang Qu, chief operating officer and co-founder of NGGT, about the company’s approach to gene therapies, how it is leveraging its platform technologies to cost-effectively accelerate development of these therapies, and the pipeline it is building.

… continue reading

520 episoder

#Health Industry #Business #Health Care #Rare Disease #RARECast #Health and Well-Being

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