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Fostering the Next Generation of Antisense Oligonucleotide Therapies

Antisense oligonucleotides (ASOs) have faced their share of challenges, but a combination of research advances and a market ripe for innovation is setting this drug class up for a resurgence. Antisense oligonucleotide (ASO) a class of drugs that works by targeting RNA to alter gene expression and control aberrant proteins in ways that traditional drugs can’t. ....

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Utility of Antisense Oligonucleotides (ASOs) in Next-Generation Biotherapeutics

Utility of Antisense Oligonucleotides (ASOs) in Next-Generation Biotherapeutics
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Paul Zamecnik , Mary Stephenson , Kim Lennox , Stanley Crooke , Fomivirsen Vitravene , Oxford University , Nucleic Acids Research , Lorem Foundation , Oxford University Press , Ionis Pharmaceuticals , Lasker Award ,

A new, more diverse human genome offers hope for rare genetic diseases

The first draft of the pangenome is based on the full genetic blueprints of 47 people from around the world ....

United States , University Of Washington , Walt Disney World , Kansas City , New Zealand General , New Zealand , Medstar Georgetown University Hospital , District Of Columbia , Papua New Guinea , Andrew Futreal , Seth Berger , Melissa Evans , Rose Gelineau , Kyle Moser , Eimear Kenny , Jean Baptiste Le Pichon , Teresa Cruz Steele , Carolyn Van Houten , Cornell Steele , Evan Eichler , Eric Green , Tom Curran , Stanley Crooke , Celia Steele , National Institutes Of Health Undiagnosed Diseases Program , National Institutes Of Health Intramural Sequencing Center ,

They created a drug for Susannah. What about millions of others?

Susannah Rosen, 8, spent much of her childhood in hospitals in New York City as doctors documented the gradual loss of her ability to stand, walk and see. ....

New York , United States , United Kingdom , Columbia University , La Jolla , David Corey , Stanley Crooke , Julia Vitarello , Timothy Yu , Jennifer Bain , Rosanne Crooke , Susannah Rosen , Wendy Chung , Jeff Milton , Sally Jackson , Mila Makovec , Issi Rozen , Luke Rosen , Drug Administration , Ionis Pharmaceuticals , New York City , Google Ventures , Boston Children , Newyork Presbyterian Morgan Stanley Children , Texas Southwestern Medical Center , Neurological Disorder ,

They Created a Drug for Susannah. What About Millions of Other Patients?

Scientists have made rapid progress in customizing drugs for ultrarare diseases. The hard part now is making such treatments on a large scale. ....

New York , United States , Columbia University , United Kingdom , David Corey , Stanley Crooke , Julia Vitarello , Timothy Yu , Jennifer Bain , Rosanne Crooke , Susannah Rosen , Wendy Chung , Jeff Milton , Sally Jackson , Mila Makovec , Issi Rozen , Luke Rosen , Drug Administration , Ionis Pharmaceuticals , New York City , Google Ventures , Boston Children , Newyork Presbyterian Morgan Stanley Children , Texas Southwestern Medical Center , Genetics And Heredity , Rare And Orphan Diseases ,