Oligonucleotides News and Research

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Oligonucleotides are short sequences of nucleotides (RNA or DNA), typically with twenty or fewer bases. Automated synthesizers allow the synthesis of oligonucleotides up to 160 to 200 bases.

New evidence supports the use of antisense oligonucleotides as feasible therapeutic strategy for MDS

Many cognitive neurodevelopmental disorders are a result of too many or too few copies of certain genes or chromosomes.

5 Mar 2021

FDA approval provides targeted treatment option for patients with rare DMD mutation

Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Exons are pieces of DNA that provide information for making proteins in a person's genome).

25 Feb 2021

mRNA Therapeutics – Analytical and Bioanalytical Characterization

NewsMed interviews Anette Bak from AstraZeneca about her research on developments in mRNA therapeutics that she will present at Pittcon 2021.

From Pittcon 23 Feb 2021

Researchers develop ultra-small nanomedicines to target intractable cancers

Ultra-small nanomedicines of approximately 18 nm were fabricated by dynamic ion-pairing between Y-shaped block copolymers and nucleic acid drugs, such as siRNA and antisense drugs.

19 Jan 2021

Replacing the RNA strand with DNA may enhance efficacy of ASO-based drugs

Antisense oligonucleotides (ASO) hold great promise for pharmacotherapy. Now, researchers at Tokyo Medical and Dental University (TMDU) and Ionis Pharmaceuticals, advancing their earlier work on a heteroduplex oligonucleotide (HDO) model, have demonstrated augmentation of ASO-based drugs by replacing the RNA strand with DNA.

5 Jan 2021

A paper-based sensor for detecting COVID-19

News-Medical talks to Dipanjan Pan about the development of a paper-based electrochemical sensor that can detect COVID-19 in less than five minutes.

5 Jan 2021

Research shows how remdesivir inhibits SARS-CoV-2 replication

In a recent bioRxiv preprint, a research team from Germany, Goran Kokic et al. study the molecular mechanism of remdesivir-induced stalling of RdRp - using synthetic RNA chemistry, biochemistry, and cryo-electron microscopy.

2 Nov 2020

Research projects receive BTCA grants to develop novel drugs for various diseases

Five research projects with exceptional promise to deliver new life-changing and health-altering therapies have received the inaugural Blavatnik Therapeutics Challenge Awards (BTCA) at Harvard Medical School.

30 Oct 2020

Inhibition of SARS-CoV-2 with antisense oligonucleotides

A part of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) viral RNA is highly conserved. Researchers have used this as a target for designing antisense oligonucleotide gapmers to cleave the RNA and disrupt virus replication. Their research is published as a preprint on the online server bioRxiv in September 2020.

22 Sep 2020

Serum protein shows therapeutic value against severe COVID-19-like features in mouse model

A new study published on the preprint server bioRxiv* in August 2020 reports the disease-mitigating effects of a serum protein called serum amyloid P (SAP) in mice exposed to a peptide that mimics the clinical signs of severe COVID-19 in humans.

30 Aug 2020

RNA-based therapeutic approach holds promise as potential cure for hereditary diseases

ETH Zurich scientists have developed an RNA molecule that can be used in bone marrow cells to correct genetic errors that affect protein production.

18 Aug 2020

FDA approves new treatment option for patients with rare Duchenne muscular dystrophy mutation

Today, the U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

12 Aug 2020

Large-scale analysis of Alport syndrome reveals the effectiveness of existing treatments

A large-scale analysis of the clinical characteristics of Alport syndrome in Japanese patients has revealed that the effectiveness of existing treatment with ACE inhibitors and/or angiotensin receptor blockers (RAS inhibitors) varies depending on the type of mutation in the syndrome's causal gene (COL4A5).

7 Aug 2020

Picomolar inhibitors to SARS-CoV-2 proteins

Now, a new study published on the preprint server bioRxiv in August 2020 reports the development of mini-inhibitors based on computational design, that are suitable for such topical use.

4 Aug 2020

Researchers develop new therapeutic approach to treat CLN3 Batten disease

Researchers led by a team at Rosalind Franklin University of Medicine and Science have devised a new therapeutic approach to treating a rare but deadly neurodegenerative genetic disease in children.

31 Jul 2020

Study: L-Grb2 has therapeutic efficacy in preclinical models of ovarian and uterine cancer

The cover for issue 29 of Oncotarget features, "In vivo effects of treatment with L-Grb2 in combination with anti-angiogenic therapy in an ovarian tumor model," by Lara, et al. which reported that adaptor proteins such as growth factor receptor-bound protein-2 play important roles in cancer cell signaling.

23 Jul 2020

Experimental drug for rare ALS shows promise in early clinical trial

An experimental drug for a rare, inherited form of amyotrophic lateral sclerosis (ALS) has shown promise in a phase 1/phase 2 clinical trial conducted at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston and other sites around the world and sponsored by the pharmaceutical company Biogen Inc.

8 Jul 2020

Developing Vaccines and Therapeutics by Exploiting Structure in Spherical Nucleic Acids

An interview discussing the use of spherical nucleic acids (SNAs) and what they show about the importance of structure in pharmaceutical development.

From Pittcon 2 Jul 2020

Researchers develop a potential treatment approach for Pelizaeus-Merzbacher disease

A team led by Case Western Reserve University medical researchers has developed a potential treatment method for Pelizaeus-Merzbacher disease (PMD), a fatal neurological disorder that produces severe movement, motor and cognitive dysfunction in children.

1 Jul 2020

Researchers develop potential new therapy for common form of muscular dystrophy

Researchers have designed a potential new treatment for one of the most common forms of muscular dystrophy, according to a new study published today in the Proceedings of the National Academy of Sciences.

29 Jun 2020