Inherited Retinal Disorder: In Vivo CRISPR Therapy Provides Vision Improvements

July 16th, 2024|Categories: Perspectives on Current Science|

Inherited retinal disorders (IRDs) are the leading cause of visual impairment, affecting approximately 1 in 2,000 people of all ages worldwide (1). There are many types of IRDs, each caused by a gene variant that affects how the retina functions, with one of the ...

Application of hydrophobically conjugated siRNAs for the treatment of dermatological and muscular diseases

July 11th, 2024|Categories: Past Webinars|

Date: July 11, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Application of hydrophobically conjugated siRNAs for the treatment of dermatological and muscular diseases Description: This webinar will focus on siRNA delivery to skin and muscle using hydrophobic ...

2023 OTS Paper of the Year Spotlight: A Framework for Individualized Splice-Switching Oligonucleotide Therapy

July 6th, 2024|Categories: Past Webinars|

Date: August 22, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: 2023 OTS Paper of the Year Spotlight: A Framework for Individualized Splice-Switching Oligonucleotide Therapy Description: Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of ...

Branching into RNAi

June 13th, 2024|Categories: Past Webinars|

Date: June 13, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Branching into RNAi Description: Our research focuses on the synthetic short-interfering RNAs (siRNAs) that silences the oncogenic mRNA expression of the Glucose Regulated Proteins (GRPs) leading to ...

President’s Pick Article – June 2024

June 12th, 2024|Categories: Presidents Pick|

Another stellar review article summarizing the field now published in Nucleic Acid Therapeutics. The review in Nucleic Acid Therapeutics by Jillian Belgrad and colleagues discusses the significant progress and future potential of nucleic acid-based therapies (NATs). It highlights the historical milestones, including the approval of ...

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