Korro Bio, Inc.’s Post

Did you hear? #Natera has over 200 peer-reviewed publications! And approximately 40 of those publications feature Natera’s organ health products, including data from ProActive, the largest prospective donor-derived cfDNA study in kidney transplantation, and data from RenaCARE, a large, multisite, real-world, prospective study assessing the frequency and impact of genetic testing in chronic kidney disease. Way to go, team! #Natera https://ow.ly/IZ1T30sFnyq

Did you hear? #Natera has over 200 peer-reviewed publications! And approximately 40 of those publications feature Natera’s organ health products, including data from ProActive, the largest prospective donor-derived cfDNA study in kidney transplantation, and data from RenaCARE, a large, multisite, real-world, prospective study assessing the frequency and impact of genetic testing in chronic kidney disease. Way to go, team! #Natera https://ow.ly/3EQK30sFnNs

Laboratory testing for acute myeloid leukemias (AMLs) includes morphologic evaluation of bone marrow and immunophenotypic, cytogenetic, and molecular genetic testing. Visit the recently updated ARUP Consult® Acute Myeloid Leukemia topic to learn more about the use of laboratory tests in AML classification, diagnosis, prognosis, and therapeutic decision-making. https://ow.ly/4YYV50SSpyg

Genomic medicine in GI disorders: Discussing the role of genetic testing and biomarker profiling in predicting disease, #prognosis, and treatment response in conditions such as colorectal cancer and familial polyposis #syndromes. #Pharmacogenomics: Highlighting the impact of genetic variations on drug metabolism and efficacy, and the potential for personalized medication regimens in inflammatory bowel disease and #hepatitis C For more details: https://lnkd.in/gUM3caRJ #gastroentrology #gastrocongress2024 #Hepatology #confrence #liverdisease #alimentarycanal

In this episode of “Answers From the Lab,” listen to Bobbi Pritt, M.D., chair of the Department of Laboratory Medicine and Pathology at Mayo Clinic, and Bill Morice, M.D., Ph.D., CEO and president of Mayo Clinic Laboratories, discuss the recent approval of two gene therapies for sickle cell disease, and bioethical and accessibility considerations of innovative treatments like gene-editing therapies. https://bit.ly/3GRYv1E

A new study of Fortea et al. analyzing large multicenter cohorts of subjects with #AlzheimerDisease biomarkers confirmed that #APOE4 homozygosity is a major genetic cause of AD, accounting for 2% of the overall population (!) and 15% of all AD cases. Thus, APOE4 represents a genetically validated drug target for AD. https://lnkd.in/e9XKM-ia Next, how to drug APOE4?

#transcript Alex Nemiroff, General Counsel at Praxis Precision Medicines, focuses on developing life-altering treatments for pediatric patients with epilepsy, particularly those with genetic epilepsies known as Developmental Epileptic Encephalopathies- DEEs.  Praxis targets specific gene mutations SCN2A and SCN8A with therapies that precisely target the source of the disease. Lead drug candidate PRAX-562 is a precision small-molecule treatment that inhibits seizure-causing activity, and PRAX-222 is a type of RNA therapy that shows potential for complete disease modification. #Praxis #Epilepsy #PediatricEpilepsy #SCN2A #SCN8A #CNS praxismedicines.com https://lnkd.in/gsQQqjMY

Are you attending the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore this week? Check out our poster (#596), "Using a Semi-mechanistic Model to Predict Efficacious Dosing Regimens and Optimal Drug Design Properties for a LNP-delivered mRNA UGT1A1 Replacement Therapy in Crigler-Najjar Syndrome Type 1 Patients," in the Liver Genetic Diseases Poster Session on Wednesday, May 8th! Learn more at: https://lnkd.in/dfEMJWxi #asgct #cellandgenetherapy #genetherapy #mechanisticmodeling #drugdesign #geneticdiseases

[INTERNATIONAL CRIGLER-NAJJAR DAY] 🌻This Friday, June 21, our teams are supporting #CriglerNajjarDay, an worldwide day dedicated to Crigler-Najjar syndrome, a rare genetic liver disease. It is characterized by the abnormal accumulation in all body and brain tissues of bilirubin, a yellow-pigmented substance produced by the liver, which can lead to severe neurological damage and death. Only phototherapy, lasting from 10 to 12 hours a day, remains the mainstay of long-term  treatment for patients suffering from this syndrome and thus enables  bilirubin levels to be reduced, but it considerably impairs patients' quality of life. 💙An international gene therapy clinical trial, sponsored by Genethon, began in 2018. The first results published in 2023 in the New England Journal of Medicine demonstrated the efficacy of this #GeneTherapy treatment designed by the «Immunology and liver diseases team» led by Giuseppe Ronzitti. The pivotal part of the trial has been initiated enabling the inclusion children 10 years old and over. 👉 https://vu.fr/BbKwx Giuseppe Ronzitti Fanny Collaud #genethon #raredisease #clinicaltrials #Liver #GeneTherapy

Since becoming interested in ET1 back in 2016, I have wondered if certain diseases were phenotypical presentations of genetic mutations of the ECE gene, various ET receptors, or a combination of both.