How can we, humans, look at our relationship to nature differently? In season three of Going Wild, on top of stories about animals, we invite you to journey through the entire ecological web — from the tiniest of life forms to apex predators — alongside the scientists, activists and adventurers who study it. Wildlife biologist and host Dr. Rae Wynn-Grant has been studying wild animals in their natural habitats all over the world for years. Our award-winning podcast takes you inside the hidde ...
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EP 159: Engineering macrophages to tackle rare disease with Noam Baumatz of Noga Therapeutics
MP3•Home de episódios
Manage episode 447802745 series 2631947
Conteúdo fornecido por Sano Genetics. Todo o conteúdo do podcast, incluindo episódios, gráficos e descrições de podcast, é carregado e fornecido diretamente por Sano Genetics ou por seu parceiro de plataforma de podcast. Se você acredita que alguém está usando seu trabalho protegido por direitos autorais sem sua permissão, siga o processo descrito aqui https://pt.player.fm/legal.
0:00 Intro to The Genetics Podcast
01:00 Welcome to Noam and discussion of his motivation to drive rare disease forward
03:00 Noam’s daughter Noga’s experience of rare disease, including an 8 to 12 month journey to diagnosis
05:10 How the experience of his daughter receiving a genetic diagnosis motivated Noam to take change into his own hands
10:12 How meeting other families impacted by rare disease led Noam to work on gene therapy for children living with primary immune deficiencies
12:33 How Noga Therapeutics is aiming to reprogram blood stem cells and the potential impact on rare diseases
15:22 Why it’s not yet possible to use the technology to correct gain of function mutations
17:42 How Noga’s program is aiding treatment of multiple primary immune deficiencies
22:25 The techniques the company uses in XLA to modify stem cells and enable expression of a functional copy of the BTK gene
26:30 Why gene therapies don’t always provide a comprehensive solution or cure
28:10 Bridging the gap between transformative therapies and the cost of delivering drugs to very small ultra-rare disease populations
34:41 Noam’s history working in traditional Chinese medicine
39:53 The importance of thinking holistically about health, from understanding g genetic root causes to considering how genes impact whole organ systems
43:15 The collaborative nature of the rare disease community and how parents who want to use their personal experience to further research and advocacy can connect with Noam
44:37 Closing remarks
188 episódios
MP3•Home de episódios
Manage episode 447802745 series 2631947
Conteúdo fornecido por Sano Genetics. Todo o conteúdo do podcast, incluindo episódios, gráficos e descrições de podcast, é carregado e fornecido diretamente por Sano Genetics ou por seu parceiro de plataforma de podcast. Se você acredita que alguém está usando seu trabalho protegido por direitos autorais sem sua permissão, siga o processo descrito aqui https://pt.player.fm/legal.
0:00 Intro to The Genetics Podcast
01:00 Welcome to Noam and discussion of his motivation to drive rare disease forward
03:00 Noam’s daughter Noga’s experience of rare disease, including an 8 to 12 month journey to diagnosis
05:10 How the experience of his daughter receiving a genetic diagnosis motivated Noam to take change into his own hands
10:12 How meeting other families impacted by rare disease led Noam to work on gene therapy for children living with primary immune deficiencies
12:33 How Noga Therapeutics is aiming to reprogram blood stem cells and the potential impact on rare diseases
15:22 Why it’s not yet possible to use the technology to correct gain of function mutations
17:42 How Noga’s program is aiding treatment of multiple primary immune deficiencies
22:25 The techniques the company uses in XLA to modify stem cells and enable expression of a functional copy of the BTK gene
26:30 Why gene therapies don’t always provide a comprehensive solution or cure
28:10 Bridging the gap between transformative therapies and the cost of delivering drugs to very small ultra-rare disease populations
34:41 Noam’s history working in traditional Chinese medicine
39:53 The importance of thinking holistically about health, from understanding g genetic root causes to considering how genes impact whole organ systems
43:15 The collaborative nature of the rare disease community and how parents who want to use their personal experience to further research and advocacy can connect with Noam
44:37 Closing remarks
188 episódios
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