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CSHL Stories and Media archive for Tag: RNA therapeutics

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Krainer named Society for RNA Therapeutics board member

4/18/2023

He helped pioneer the field. It changed medicine. Now, he joins a group of renowned scientists and physicians aiming to take it to the next level. Read the story »

From tragedy, a potential pediatric cancer treatment

4/12/2023

CSHL Professor Adrian Krainer, the man behind the “miracle drug” known as Spinraza®, has found a way to fight a deadly pediatric brain cancer in mice. Read the story »

A molecular machine’s secret weapon exposed

2/23/2023

A shape-shifter with a protruding arm and an appetite for unwanted RNA! CSHL biochemists identify the hidden talents of a mysterious molecule. Read the story »

A new treatment approach for cystic fibrosis

7/14/2022

Molecules called antisense oligonucleotides may help lung cells make a protein missing in people with cystic fibrosis. Read the story »

Image of dropper adding liquid to glass laboratory test tube

The promising drug duo that may improve SMA treatment

7/11/2022

Pairing Spinraza® with a second FDA-approved drug may be a new way to improve the drug’s therapeutic effects in spinal muscular atrophy patients. Read the story »

Krainer awarded Watanabe Prize in Translational Research

6/16/2022

Indiana University School of Medicine honored Krainer for his pioneering work on RNA splicing, which led to the first FDA-approved SMA therapeutic. Read the story »

Decoding how a protein on the move keeps cells healthy

5/31/2022

The Argonaute protein is a workhorse for cell regulation and CSHL researchers discovered what helps it commute from job to job. Read the story »

photo of breast cancer tumor cross section

Can cancer be treated by changing its cells?

4/11/2022

Tumors grow when cells lose their biological identity. A promising therapeutic might restore their sense of self. Read the story »

image of a mouse liver tissue with cancer tumor

Cutting off liver cancer’s nutrient supply chain

2/3/2022

CSHL researchers developed a way to interfere with the energy pathway that allows liver cancer to grow and spread. Read the story »

Editing RNA to fix protein problems in cystic fibrosis

1/27/2022

CSHL researchers found a new way to address a previously untreatable class of mutations in the cystic fibrosis gene. Read the story »

illustration of a single strand ribonucleic acid

The rise of RNA therapeutics

10/14/2021

RNA has been making waves as a new approach to prevent or treat diseases, including COVID-19 and spinal muscular atrophy. Read the story »

photo of Maria Luisa Pineda working in a lab

Envisagenics and Biogen partner for RNA splicing research

6/9/2021

CSHL spin-out company Envisagenics teams up with Biogen to advance research in RNA-based therapeutics for central nervous system diseases. Read the story »

image of cancer cell movement and metastases

Regulatory RNAs promote breast cancer metastasis

12/22/2020

A gene-regulating bit of RNA promotes breast cancer metastasis. Agents that destroy that RNA provide hope for a new drug. Read the story »

photo of Adrian Krainer and Mohammad Alinoor Rahman observing results

Mutation’s role in blood cancers revealed by ideal team-up

1/31/2020

Researchers from CSHL and MSKCC have determined how a mutation implicated in many blood cancers reduces the proteins that make healthy blood cells. Read the story »

graphic of title card from Mutations in Blood Cancers video

Mutation’s role in blood cancers revealed by ideal collaboration