Oligonucleotide News and Research

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Cell-penetrating nanoparticles can efficiently transport oligonucleotide drugs into cells

Therapeutic oligonucleotide analogs represent a new and promising family of drugs that act on nucleic acid targets such as RNA or DNA; however, their effectiveness has been limited due to difficulty crossing the cell membrane.

31 Jan 2015

ALS Association announces research funds to further understand genetic cause of ALS

The ALS Association is pleased to announce the award of $326,662 in research funds to expand ongoing natural history studies in order to further understand the most common genetic cause of ALS, in preparation for clinical trials in those whose disease is affected by this gene.

14 Dec 2014

Study: Injectable 3D vaccines could help prevent cancer, infectious disease

One of the reasons cancer is so deadly is that it can evade attack from the body's immune system, which allows tumors to flourish and spread. Scientists can try to induce the immune system, known as immunotherapy, to go into attack mode to fight cancer and to build long lasting immune resistance to cancer cells.

9 Dec 2014

Studies presented at ASH meeting compare new, standard-of-care treatments for blood clots

Studies presented at the 56th American Society of Hematology Annual Meeting and Exposition compare new and standard-of-care treatments for blood clots and further illuminate clot risks in vulnerable populations, such as cancer patients.

8 Dec 2014

New light-triggered strategy can provide accurate control over aptamer-based therapeutics

While aptamers—single-stranded nucleic acids—offer promise as drug-delivery tools for cancer treatment, it has proven difficult to get them to tumor cells at therapeutic levels.

4 Dec 2014

Researchers explore how to turn on the activity of paternal gene

Most genes are inherited as two working copies, one from the mother and one from the father. However, in a few instances, a gene is imprinted, which means that one copy is silenced. This is called genomic imprinting. If the active copy is mutated, then disease results, even though the silenced gene copy may be normal.

2 Dec 2014

Isis Pharmaceuticals, AstraZeneca partner to develop new delivery methods for antisense oligonucleotides

AstraZeneca and Isis Pharmaceuticals Inc. today announced a strategic alliance to discover and develop novel delivery methods for antisense oligonucleotides.

14 Nov 2014

New consensus guidelines for toxicity testing of oligonucleotide-based therapeutics

Oligonucleotide-based therapeutics present unique challenges when it comes to testing their potential to cause reproductive and developmental harm. New consensus guidelines for toxicity testing that take into consideration the combined chemical and biological characteristics of these novel biopharmaceuticals are presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers.

31 Oct 2014

Regulus Therapeutics demonstrates human proof-of-concept with microRNA therapeutic

Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that it has demonstrated human proof-of-concept with a microRNA therapeutic from an ongoing clinical study evaluating RG-101, a wholly-owned, GalNac-conjugated anti-miR targeting microRNA-122 (“miR-122”), for the treatment of hepatitis C virus infection (“HCV”).

22 Oct 2014

Identifying infections rapidly: an interview with Dr. David J. Ecker

Current methods for diagnosing infectious diseases are based on the 150-year-old culture method, where physicians collect a sample of a patient’s tissue, such as blood, mucus or urine, and transfer it onto media bottle to allow the pathogens to grow.

From Abbott Diagnostics 20 Oct 2014

TSRI scientists awarded DARPA grant to build artificial immune system

Scientists from both campuses of The Scripps Research Institute have been awarded a total of $7.9 million from the Defense Advanced Research Projects Agency of the U.S. Department of Defense.

25 Sep 2014

Phase IIa clinical trial data of ATL1102 in MS patients published in journal Neurology

Antisense Therapeutics is pleased to report the publication of previously generated Phase IIa clinical trial data on ATL1102 in the medical journal Neurology.

22 Sep 2014

Regulus begins ATHENA study to monitor changes in renal function in Alport syndrome patients

Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today it has initiated its ATHENA natural history of disease study in patients with Alport syndrome, a life-threatening genetic kidney disease with no approved therapy.

19 Sep 2014

MU researchers make new breakthrough in spinal muscular atrophy drug

According to recent studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over time.

16 Sep 2014

Researchers find method to repair gene mutation causing autoimmune deficiency disease

Researchers at Karolinska Institutet in Sweden have found a method to repair the gene mutation causing agammaglobulinemia, an autoimmune deficiency disease that almost exclusively affects boys and in which the body lacks the ability to produce immunoglobulins (gamma globulin).

14 Aug 2014

Oligonucleotide News and Research

Cell-penetrating nanoparticles can efficiently transport oligonucleotide drugs into cells

ALS Association announces research funds to further understand genetic cause of ALS

Study: Injectable 3D vaccines could help prevent cancer, infectious disease

Studies presented at ASH meeting compare new, standard-of-care treatments for blood clots

New light-triggered strategy can provide accurate control over aptamer-based therapeutics

Researchers explore how to turn on the activity of paternal gene

Isis Pharmaceuticals, AstraZeneca partner to develop new delivery methods for antisense oligonucleotides

New consensus guidelines for toxicity testing of oligonucleotide-based therapeutics

Regulus Therapeutics demonstrates human proof-of-concept with microRNA therapeutic

Identifying infections rapidly: an interview with Dr. David J. Ecker

TSRI scientists awarded DARPA grant to build artificial immune system

Phase IIa clinical trial data of ATL1102 in MS patients published in journal Neurology

Regulus begins ATHENA study to monitor changes in renal function in Alport syndrome patients

MU researchers make new breakthrough in spinal muscular atrophy drug

Researchers find method to repair gene mutation causing autoimmune deficiency disease

New chromatin immunoprecipitation sequencing kit developed by Porvair Sciences

Splice-switching oligonucleotide drugs alter editing of gene transcript

Study of cancer radiation therapy shows that DNA building blocks are susceptible to fragmentation

Marginal division of striatum plays similar function in learning and memory to hippocampus

Scientists release recommendations for evaluating pharmacological safety of oligonucleotide therapeutics

Exploring the G:Box and for high-quality imaging

Shaping the Future of Neuroscience: A Conversation with Atlas Antibodies on the MolBoolean™ and the Impact of SfN 2024

How the Arts Reshape Brain Function: Susan Magsamen on the Future of Neuroaesthetics

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