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alnylam.com

Learn how RNAi therapeutics work and utilize small interfering RNA (siRNA) to silence genes that cause, or contribute to disease.

modernatx.com

Learn how we’re changing the world of medicine. Discover career opportunities, our product pipeline, and browse media resources. Meet Moderna.

scge.mcw.edu

We utilized novel amphiphilic shuttle peptides to deliver base editor ribonucleoprotein (RNP) into the airways to edit airway epithelial cells (CCR5 locus) of rhesus monkeys. The Cas9-ABE8e RNP and shuttle peptides S10 or FSD315 were aerosolized into the rhesus monkey trachea. Seven days later, tissues were obtained and dissected, and airway epithelia collected from the trachea, mainstem, and segmental bronchi using cytology brushes. DNA was extracted from epithelial cells and subjected to high-throughput sequencing. Using the FSD315 shuttle peptide and Cas9-ABE8e, we achieved a mean editing efficiency of 2.8% at the CCR5 locus in airway epithelial cells (range 0.02 – 5.3%) depending on the anatomic region sampled. To visualize the biodistribution of the RNPs within the respiratory tract and in specific cell types, we delivered a Cy5-fluorescent peptide fused to a nuclear localization signal (NLS-Cy5) using the S10 peptide. The lungs were obtained 1 and 2 hours post-delivery, fixed, and examined by microscopy. Epifluorescence and confocal microscopy documented an effective intra-nuclear delivery of NLS-Cy5 into epithelial cells throughout the respiratory tract, including large, medium, and small airways, and alveolar regions. Ongoing analyses will identify the NLS-Cy5-positive epithelial cell types using co-localization with fluorescently-labeled antibodies. In summary, using a rhesus monkey model, following a single delivery of adenine base editor RNPs to the airways in a clinically relevant manner we achieved up to 5.3% editing efficiency of the CCR5 locus in airway epithelia, a level considered therapeutically relevant in cystic fibrosis.

ebresearch.org

For the Deshe Family, the mission is clear, “We would love to see this disease gone, and we want to be a part of helping eradicate it—forever.” Ari Deshe, the founder and chairman of Safe Auto...

debra.org

debra of america is a non-profit organization providing free programs & eb support services to those living with epidermolysis bullosa. make a donation and help fund research for a cure.

vyriad.com

Read the latest about our team at Vyriad in the news.

elevidys.com

Sign up to receive information and updates about ELEVIDYS (delandistrogene moxeparvovec-rokl).

arrowheadpharma.com

Watch and listen to a webinar on real-life insights about communicating with healthcare providers, coping with stress, supporting mental health and navigating social realities of FCS.

miltenyibiotec.com

miltenyi biotec offers research- and clinical-grade products for sample preparation, cell separation, flow cytometry, and cell culture applications. | usa