Tenaya Therapeutics’ Post

Gene mutations can change the instructions the body uses to make proteins. The #MYBPC3 gene guides protein production needed for the heart to pump as expected. A mutation (variation) in this gene can lead to #HypertrophicCardiomyopathy. #HCM #HeartDisease #GeneTherapy #Genetics

New AusPAR for Hemgenix (etranacogene dezaparvovec). Hemgenix is a gene therapy for the treatment of adults with haemophilia B (congenital factor IX deficiency). Hemgenix is a modified virus (that is harmless to humans) that contains a healthy copy of the gene for clotting Factor IX. This gene is delivered to the liver by the virus where it is able to instruct cells to produce factor IX, supporting natural blood-clotting ability. Read more: https://lnkd.in/gcsfe9js

Tempo and mode of gene expression evolution in the brain across primates Explores influence of gene expression on primate brain evolution. Identifies novel candidate genes for the study of genetic regulation of brain evolution. https://lnkd.in/gid6TyxT

New AusPAR for Hemgenix (etranacogene dezaparvovec). Hemgenix is a gene therapy for the treatment of adults with haemophilia B (congenital factor IX deficiency). Hemgenix is a modified virus (that is harmless to humans) that contains a healthy copy of the gene for clotting Factor IX. This gene is delivered to the liver by the virus where it is able to instruct cells to produce factor IX, supporting natural blood-clotting ability. Read more: https://lnkd.in/gcsfe9js

Adeno-Associated Viruses are important tools for therapeutic gene delivery, successfully treating diseases where a gene is missing, required editing or silencing! Our technote on AAV and Gene Therapy discusses the applications of AAV in therapy and its current challenges. BPS Bioscience provides researchers with a set of AAV tools that can support new discoveries. #aav #gene #drusdiscovery https://hubs.ly/Q02FV6yT0

DNA holds the blueprint of life, but gene regulation is what makes it dynamic! From transcription factors to mRNA, gene expression is the key to how cells function and evolve. #Genetics #GeneRegulation #Biotech #DNA #MolecularBiology #ScienceBreakthroughs #Endocrinology2025

In addition to cleaving RNA and single-stranded DNA (ssDNA) by transesterification or a blend of hydrolytic and transesterification reactions, natural ribozymes have now been found to catalyze the hydrolytic cleavage of ssDNA, suggesting that such ribozymes hold promise as alternatives to protein-based nucleases in gene therapies. More Reading King, M.B., Lapinaite, A. RNA-based programmable DNA cleavage. Nat Chem Biol(2024). https://lnkd.in/g2zKvTGb

Understanding therapies that look to correct the inherited changes or mutations in DNA of the genome of individuals with a #raredisease can be confusing. This #IRDiRC sponsored paper provides an overview of the different types of gene(tic) approaches to such therapy development.

Creative Biogene offers diverse dsRNA controls as reliable positive controls for precise dsRNA assays. These controls empower researchers to induce RNA interference, study gene functions, and facilitate gene silencing studies. https://lnkd.in/gq2mzPVK

This fascinating article demonstrates the power of gene editing. Researchers at the University of Helsinki and HUS Helsinki University Hospital have succeeded in correcting a gene defect that causes a hereditary liver disease and its adverse effects on cells. While this is a non-mito metabolic condition, there are similarities in traditional approaches to management. Read more here: https://lnkd.in/e88Nih6y