An insider’s view into the preclinical development of an approved exon skipping drug

October 21st, 2022|Categories: Past Webinars|

Time: 11am - 12pm EST Date: November 3, 2022 Description: Dr. Arechavala works on developing new drugs for neuromuscular disorders. As there are not many for these rare diseases, neuromuscular disorders have been at the forefront of the clinical development of RNA based ...

Site-specific detection of mutagenic DNA damage with an artificial nucleotide

October 21st, 2022|Categories: Past Webinars|

Time: 11am - 12pm EST Date: October 27, 2022 Description: Incorporating nucleotides with altered structures that are derived synthetically is a strategy that has been used widely to interrogate biological processes involving DNA. Particularly, synthetic nucleotides able to stabilize damaged over undamaged templates ...

Eplontersen May Soon Provide Another Safe, Effective Treatment for People Diagnosed with ATTR

September 23rd, 2022|Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

People who have been diagnosed with hereditary transthyretin amyloidosis (ATTRv) may soon have the option of choosing another safe, effective therapy, one that significantly improves quality of life, according to patients who received it in the clinical trial. ATTRv is a progressive, debilitating, ...

2022 Paper of the Year Award – Basic Research Category

September 19th, 2022|Categories: Basic Featured Paper of the Year, Paper of the Year Award|

Tetsuya Nagata, Takanori Yokota, et al, Nature Biotechnology (2021). https://doi.org/10.1038/s41587-021-00972-x Takanori Yokota, MD, PhD Tokyo Medical and Dental University Biography: Dr. Yokota graduated from Tokyo Medical and Dental University in 1984 and completed his Residency and Fellowship in Musashino Red Cross Hospital ...

2022 Paper of the Year Award – Basic Research Category

September 19th, 2022|Categories: Basic Featured Paper of the Year, Paper of the Year Award|

Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates Kirk M. Brown, Martin A. Maier, Vasant Jadhav, et al, Nature Biotechnology (2022). https://doi.org/10.1038/s41587-022-01334-x Vasant Jadhav, PhD Alnylam Pharmaceuticals Biography: Vasant Jadhav is a Senior Vice President of Research at Alnylam Pharmaceuticals, where he ...

2022 Award for Science Outreach Winner Angela Messmer-Blust, PhD

September 19th, 2022|Categories: Award for Science Outreach|

Angela Messmer-Blust, PhD Associate Professor, RTI, UMass Chan Medical School Biography: Dr. Angela Messmer-Blust studied cell motility during her graduate studies at the University of Toledo where she received her Ph.D. (2009). She began her postdoctoral research at the CardioVascular Institute at Beth Israel ...

2022 Award for Patient Advocacy Winner Daniel O’Reilly, PhD

September 19th, 2022|Categories: Award for Patient Advocacy|

Daniel O’Reilly, PhD RTI, UMass Chan Medical School Biography: Daniel O’Reilly is currently a Post-Doctoral Associate in the Lab of Professor Anastasia Khvorova in the RNA Therapeutics Institute at the University of Massachusetts Chan Medical School. Dan earned his Ph.D. in Chemistry under the ...

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