RNA therapeutics Archives - Cold Spring Harbor Laboratory

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Andrea Schorn zooms in on small RNAs in the cell

April 29, 2019

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

Therapeutic RNA corrects splicing defect that causes familial dysautonomia

April 30, 2018

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

New method identifies splicing biomarkers for liver cancer

March 2, 2018

Researchers have developed a method for identifying splicing-based biomarkers for liver cancer

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

December 24, 2016

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

One experiment: A drug for SMA visibly repairs defective cells

December 1, 2016

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

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

September 27, 2016

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

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

April 20, 2016

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

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

December 22, 2015

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

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

November 3, 2012

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

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

August 15, 2012

Researchers find novel method of making animal models for human genetic disease research.

CSHL announces collaboration to speed development of new cancer therapies

June 13, 2012

Short hairpin RNA collection applied to advanced mouse models aims to accelerate drug target validation.

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

October 5, 2011

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

Collaboration column: SMA therapeutics and potential drug target for schizophrenia

March 11, 2011

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

New method powerfully boosts efficiency of RNA interference (RNAi) in shutting down genes

February 24, 2011

CSHL-led team finds an accurate, scalable way to identify potent RNAi triggers for biological studies and for RNA therapeutics.

CSHL researchers demonstrate efficacy of antisense therapy for spinal muscular atrophy

July 12, 2010

Researchers look into the use of chemically-modified pieces of RNA called antisense oligonucleotides to treat SMA.

CSHL Story archive for Tag: RNA therapeutics