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Genentech announces clinical data from studies investigating risdiplam in spinal muscular atrophy

Genentech, a member of the Roche Group, announced today interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam in spinal muscular atrophy.

3 Oct 2018

Study highlights role of non-mutational mechanism in myotonic dystrophy, other types of arrhythmias

A team of researchers at Baylor College of Medicine reveals new insights into what can be causing life-threatening cardiac-related events in myotonic dystrophy type 1.

28 Sep 2018

Researchers uncover molecular underpinnings of liver regeneration

In Greek mythology, Zeus punishes the trickster Prometheus by chaining him to a rock and sending an eagle to eat a portion of his liver every day, in perpetuity. It was the right organ to target - the liver has the ability to regenerate itself, though not overnight nor for eternity.

27 Sep 2018

Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

Study could pave way for new therapies to reverse aging in human cells

Research has shed new light on genetic processes that may one day lead to the development of therapies that can slow, or even reverse, how our cells age.

14 Sep 2018

Researchers predict how splicing errors impact disease risk

No one knows how many times in a day, or even an hour, the trillions of cells in our body need to make proteins.

31 Aug 2018

New CRISPR technology may help eliminate mutated gene sequence

In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell's internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building.

17 Aug 2018

ETH scientists discover new cancer-specific molecular changes in tumor tissues

A research team led by Gunnar Raetsch, Professor of Biomedical Informatics at ETH Zurich, has evaluated the largest set of genetic data in cancer medicine: The Cancer Genome Atlas in the United States.

9 Aug 2018

New compounds can reverse key aspects of aging of human cells

Key aspects of the aging of human cells can be reversed by new compounds developed at the University of Exeter, research shows.

7 Aug 2018

Inhibition of NOVA1 gene helps prevent growth of lung cancer tumors

Researchers have identified a gene that when inhibited or reduced, in turn, reduced or prevented human non-small cell lung cancer tumors from growing.

7 Aug 2018

Alternative splicing plays vital role in adult muscle mass maintenance

Despite the importance that changes in muscle mass have in aging, overall body metabolism and in chronic disease, we still don't fully understand the mechanisms that contribute to the maintenance of adult muscle mass.

11 Jul 2018

Study finds that several existing anti-cancer drugs may work in part by binding to RNA

Bolstering the notion that RNA should be considered an important drug-discovery target, scientists at Scripps Research have found that several existing, FDA-approved anti-cancer drugs may work, in part, by binding tightly to RNA, the regulators of the basic activities of life within cells.

30 Jun 2018

Blocking viral-host interaction slows down flu virus replication, study shows

Influenza A (flu A) hijacks host proteins for viral RNA splicing and blocking these interactions caused replication of the virus to slow, according to new research published in Nature Communications by Kristin W. Lynch, PhD, chair of the department of Biochemistry and Biophysics in the Perelman School of Medicine at the University of Pennsylvania, and doctoral student Matthew Thompson.

25 Jun 2018

Unusual cell-to-cell communication in glioblastoma promotes aggressiveness and therapy resistance

A surprising form of cell-to-cell communication in glioblastoma promotes global changes in recipient cells, including aggressiveness, motility, and resistance to radiation or chemotherapy.

22 Jun 2018

Scientists discover a mechanism through which HIV determines the fate and function of cells

A stem cell is one with infinite possibilities. So, for decades, scientists have puzzled over how the cell chooses to keep being a stem cell and continue dividing, or specialize into a specific cell type, like a heart or brain cell.

11 May 2018

Scientists discover how potential new drug targets spinal muscular atrophy

Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic mutation and stop its progression.

8 May 2018

Researchers discover how key brain receptor can function in hostile environment

During a stroke or an epileptic seizure, neurons in affected parts of the brain fire at an abnormally rapid rate.

7 May 2018

Therapeutic RNA corrects error in genetic processing that leads to familial dysautonomia

Scientists at Cold Spring Harbor Laboratory have identified a therapeutic RNA molecule that corrects the error in genetic processing that leads to familial dysautonomia, a rare inherited neurodegenerative disorder.

30 Apr 2018

Investigational drug may help increase protein levels in babies with spinal muscular atrophy

A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy. The open-label study is released today and will be presented at the American Academy of Neurology's 70th Annual Meeting in Los Angeles, April 21 to 27, 2018.

19 Apr 2018

CNIO researchers determine structure of protein complex related to cell survival

A team from the Spanish National Cancer Research Centre has determined for the first time the high-resolution structure of a complex (R2TP) involved in key processes for cell survival and in diseases such as cancer.

16 Apr 2018