Collaborative Research LED by Investigators at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Defines a Novel Approach to Undersastanding How Certain Proteins Called Transact Factors Determine which genetic programs will drive cell growth and maturation. The study is Published in the journey Science,

The method, Called “Perturb-Multiome,” Uses Crispr to Knock out the Function of Individual Transportation Factors Across Many Blood Cells at Once. The Researchers then Perform Single-Cell Analyses on Each Cell to Measure The Effects of the Editing, Including Identtifying which genes have been on Epigenetic markers). The team applied this tool to Immature blood cells to identify the important transcription factors –and dna regions that code for them -in struggly affect how blood cells devallop.

They discovered that many of the DNA regions they identified as important for driving blood Cell production are also also also records known to Harbor mutations linked to Blood disorders. The DNA Regents they identified as important obscupy less than 0.3% of the genome, but they explain a disproportionatete

In earlier work, Investigators from this team and other collaborators used genome-with association studies to identify the transcription factorsible for switching of Fetal Hemoglobin AFFTALBIN AFFTALBIN AFFTALBIN AFFER Groundwork for the Development of Gene Therapy for Sickle Cell Disease and Beta Thalassemia.

This new perturb-multiome approach enables Researches Researches to Systematically Reveal How Thousands of Transcription Factor Variants Influence BLOOD CELL PRODUCTION and Influence Disease Risk, Creating, Opportunities for Finding Many More Novel Targeted Therapies for Blood Disorders.