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CSHL Stories and Media archive for Tag: RNA therapeutics

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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 »

Andrea Schorn zooms in on small RNAs in the cell

4/29/2019

Dr. Andrea Schorn joins CSHL faculty as research assistant professor. Her focus is exploring transposable elements in the genome of mammalian cells. Read the story »

A promising new strategy to combat familial dysautonomia

5/17/2018

Scientists determine how to correct the error in RNA splicing that causes familial dysautonomia, a rare inherited neurodegenerative disorder. Watch the video »

Therapeutic RNA corrects splicing defect that causes familial dysautonomia

4/30/2018

A team provides proof of concept for an RNA drug correcting an error in RNA splicing that causes familial dysautonomia (FD) Read the story »

New method identifies splicing biomarkers for liver cancer

3/2/2018

Researchers have developed a method for identifying splicing-based biomarkers for liver cancer Read the story »

FDA approval of life-saving SMA drug is hailed by its researcher-inventor at CSHL

12/24/2016

Nusinersen (Spinraza) will be used to treat young children with a severe and potentially fatal illness called spinal muscular atrophy (SMA). Read the story »

One experiment: A drug for SMA visibly repairs defective cells

12/1/2016

Professor Adrian Krainer develops a treatment for spinal muscular atrophy, the leading genetic cause of infant death. Read the story »

Non-coding portions of genome are found to play role in cancer

9/27/2016

CSHL scientists test an antisense method of targeting long noncoding RNAs overexpressed in breast cancers. Read the story »

Adrian Krainer of Cold Spring Harbor Laboratory elected to American Academy of Arts and Sciences

4/20/2016

Adrian Krainer was elected to the American Academy of Arts and Sciences. Read the story »

Unusual drug target and drug generate exciting preclinical results in mouse models of metastatic breast cancer

12/22/2015

Researchers publish preclinical data suggesting the promise of a novel drug directed against a novel target n malignant mammary tumors. Read the story »

Team uses antisense technology that exploits gene splicing mechanism to kill cancer cells

11/3/2012

Researchers find method to target the cancer cell's metabolic process to destroy it. Read the story »

CSHL-led team introduces new method to closely model diseases caused by splicing defects

8/15/2012

Researchers find novel method of making animal models for human genetic disease research. Read the story »

CSHL announces collaboration to speed development of new cancer therapies

6/13/2012

Short hairpin RNA collection applied to advanced mouse models aims to accelerate drug target validation. Read the story »

Antisense therapy delivers long-term correction of severe spinal muscular atrophy in mice

10/5/2011

Researchers report surprising results that suggest that spinal muscular atrophy might not exclusively affect the motor neurons in the spinal cord. Read the story »

Collaboration column: SMA therapeutics and potential drug target for schizophrenia

3/11/2011

CSHL's Adrian Krainer works on treatment for spinal muscular atrophy that reverses the disease's symptoms. Read the story »