National Human Genome Research Institute (NHGRI)’s Post

Hurry up, time is running out! Spread the word if you know someone affected by Sickle Cell Disease! Don't miss our upcoming webinar with Teresa Ginger Davis from Sickle Cell Thalassemia Patients Network and Farah Gaillard MSN, RN, FNP from Vertex Pharmaceuticals. They will be discussing the patient perspective on gene therapy for Sickle Cell Disease. We look forward to sharing this important conversation with you. https://bit.ly/3vH4p3O #sicklecelldisease #raredisease #patientawareness

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Today, we proudly join the global community in raising awareness for Rare Disease Day. At Kyfora Bio, we are dedicated to providing innovative solutions to pioneer advanced cell and gene therapies that offer hope to those affected by rare diseases. Our transfection reagents are used in several clinical trials globally. Together, let's continue to break barriers, drive research, and transform lives.    Read a few examples of how our transfection reagents are used in rare disease research:     Farby's Disease: https://lnkd.in/ebxbiTRH     Hemophilia: https://lnkd.in/e4wPw3t7     Huntington's Disease: https://lnkd.in/ePah_N-6     Stargardt Disease: https://lnkd.in/eQsjE6KC     #rarediseaseday #kyforabio #gene therapy #celltherapy #bioprocessing 

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May is Melanoma Awareness Month. Check out this conversation with three authors of a recent POT1 study. Come for the research recap, but stay for the discussion of gene disease validity, limited evidence genes, and the importance of clinical utility when designing and choosing multigene panels. #genetictesting #POT1 #MelanomaAwareness https://hubs.ly/Q02z5sNG0

SpCas9 has revolutionized modern medicine with groundbreaking potential for treating genetic diseases, cancer, and infectious diseases. However, as scientists push the boundaries of gene editing, it's time to explore beyond SpCas9. Expand your knowledge on the cutting-edge of genetic research and discover how these alternative #crispr nucleases could transform therapeutic applications in our blog. 🔗 👉 https://lnkd.in/dzixA7aA

Did you know that our recently announced preclinical data showed sustained upregulation of HFN4α gene expression and coordinated pre-transcriptional downregulation of multiple genes in models of liver fibrosis and inflammation? Learn more about our findings, which were presented last month at the American Association for the Study of Liver Diseases (AASLD)'s The Liver Meeting ®: https://brnw.ch/21wFfVD #TLM2023