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Study: Reducing mutated Huntington disease protein restores psychiatric impairments in mice

New research from the University of British Columbia suggests that reducing mutated Huntington disease protein in the brain can restore cognitive and psychiatric impairments in mice.

4 Oct 2018

Alnylam reports further progress on broadening the therapeutic potential of RNAi

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today further progress on the Company's platform efforts in extrahepatic delivery of novel siRNA conjugates, including central nervous system (CNS) and ocular delivery in rat and non-human primates (NHPs).

3 Oct 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Scientists create new tool to precisely control 3D architecture of linkages in drugs

When it comes to certain molecules, shape makes all the difference. The shape of limonene, for instance, a compound produced by citrus fruits, determines whether it tastes like orange juice or turpentine.

7 Aug 2018

Newer version of ALS drug may effectively treat inherited form of the disease

NIH-funded researchers delayed signs of amyotrophic lateral sclerosis in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1.

27 Jul 2018

PureTech Health collaborates with Roche to advance oral administration of antisense oligonucleotides

PureTech Health plc, a clinical-stage biopharmaceutical company developing novel medicines focused on the Brain-Immune-Gut Axis, today announced that it has entered into a multiyear collaboration with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., to advance PureTech's milk-derived exosome platform technology for the oral administration of Roche's antisense oligonucleotide platform.

20 Jul 2018

Researchers discover new approach for treatment of myotonic dystrophy

Researchers at the Translational Genomics laboratory of Valencia University and the INCLIVA health research institute have just discovered a new approach for the treatment of myotonic dystrophy, a rare and incurable neuromuscular disease which does not have specific treatment.

27 Jun 2018

Researchers employ nucleotide-based gene silencing to mitigate common ataxia

In what researchers are calling a game changer for future ataxia treatments, a new study showed the ability to turn down the disease progression of the most common dominantly inherited ataxia.

20 Jun 2018

New approach to kill specific bacteria could be alternative to antibiotics

A new approach to killing C. difficile that silences key bacterial genes while sparing other bacteria may provide a new way to treat the most common hospital-acquired bacterial infection in the United States, according to researchers.

7 Jun 2018

Alnylam achieves delivery of novel siRNAs conjugates to the central nervous system

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the Company has achieved delivery of novel small interfering RNA conjugates to the central nervous system and is planning to advance a pipeline of investigational RNAi therapeutics into clinical development.

8 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

Researchers trigger E. coli’s toxin-antitoxin system to inhibit bacterial growth

According to new research, the toxin-antitoxin (TA) system found within E. coli can be induced to inhibit the growth of the bacterium. This may have important consequences for human infection.

23 Apr 2018

Study highlights potential of ASO therapy to treat Fuchs endothelial corneal dystrophy patients

A proof-of-concept study carried out at UCL Institute of Ophthalmology with funding from Fight for Sight has highlighted the potential of a targeted antisense oligonucleotide therapy to treat Fuchs endothelial corneal dystrophy patients with a mutation in the TCF4 gene.

12 Mar 2018

New study shows advantages of DNA barcoding technique for identifying nanoparticles

The first direct comparison of in vitro and in vivo screening techniques for identifying nanoparticles that may be used to transport therapeutic molecules into cells shows that testing in lab dishes isn't much help in predicting which nanoparticles will successfully enter the cells of living animals.

5 Mar 2018

Researchers develop method to identify splicing-based biomarkers for liver cancer

Because liver cancer is particularly diverse, genetically, and prone to relapse, identifying biomarkers that can predict disease progression is a critical goal in the fight against it.

2 Mar 2018

RNA-based method cures lung tumors in mice

By turning down the activity of a specific RNA molecule researchers at Sahlgrenska Academy, Sweden, have cured lung tumors in mice by 40-50 percent.

28 Feb 2018

Scientists decode molecular structure of healthy huntingtin protein

Mutations on a single gene, the huntingtin gene, are the cause of Huntington's disease. They lead to an incorrect form of the correspondent protein.

22 Feb 2018

New medication improves motor function in children with later-onset spinal muscular atrophy

Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today in the New England Journal of Medicine.

15 Feb 2018

Scientists develop immune-stimulating injections against cancer

A team of researchers have developed two immune-stimulating injections, that removed all traces of cancer when injected directly into solid mouse tumors.

2 Feb 2018

MGH study points toward potential strategy for treating X-linked disorders

A study from a team of Massachusetts General Hospital (MGH) investigators points toward a potential strategy for treating X-linked disorders - those caused by mutations in the X chromosome - in females.

4 Jan 2018