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Genetic base editing treats sickle cell disease in mice


Broad Institute of MIT and Harvard
Sickle cell disease (SCD) is the most common deadly genetic disorder, affecting more than 300,000 newborns worldwide each year. It leads to chronic pain, organ failure, and early death in patients. A team led by researchers at the Broad Institute of MIT and Harvard and St. Jude Children’s Research Hospital has now demonstrated a base editing approach that efficiently corrects the mutation underlying SCD in patient blood stem cells and in mice. This gene editing treatment rescued the disease symptoms in animal models, enabling the long-lasting production of healthy blood cells.
The root of SCD is two mutated copies of the hemoglobin gene, HBB, which cause red blood cells to transform from a circular disc into a sickle shape setting off a chain of events leading to organ damage, recurrent pain, and early mortality. In this study, the researchers used a molecular technology called base editing to directly convert a single letter of patho ....

David Liu , Gregory Newby , Kaitly Woodard , Mitchell Weiss , Broad Institute , Harvard University , Merkin Institute Of Transformative Technologies In Healthcare , Jude Children Research Hospital , Jude Department Of Hematology , Howard Hughes Medical Institute , Jude Children , Research Hospital , Richard Merkin Professor , Merkin Institute , Transformative Technologies , Jude Department , Jonathan Yen , Thiyagaraj Mayuranathan , Cell Biology , Chronic Pain , Sickle Cell , Stem Cells , டேவிட் லியூ , கிரெகொரி புதியவர் , மிட்செல் வெயிஸ் , பரந்த நிறுவனம் ,

High-throughput system can regulate gene expression for treating sickle cell disease, beta thalassemia


High-throughput system can regulate gene expression for treating sickle cell disease, beta thalassemia
St. Jude Children s Research Hospital scientists have developed an integrated, high-throughput system to better understand and possibly manipulate gene expression for treatment of disorders such as sickle cell disease and beta thalassemia. The research appears today in the journal
Nature Genetics.
Researchers used the system to identify dozens of DNA regulatory elements that act together to orchestrate the switch from fetal to adult hemoglobin expression. The method can also be used to study other diseases that involve gene regulation.
Regulatory elements, also called genetic switches, are scattered throughout non-coding regions of DNA. These regions do not encode genes and make up about 98% of the genome. The elements have a variety of names enhancer, repressor, insulator and more but the specific genes they regulate, how the regulatory elements act together, an ....

Mitchell Weiss , Emily Henderson , Jude Children Research Hospital , Jude Children , Research Hospital , Study Corresponding Author , மிட்செல் வெயிஸ் , எமிலி ஹென்டர்சன் , ஜூட் குழந்தைகள் ஆராய்ச்சி மருத்துவமனை , ஜூட் குழந்தைகள் , ஆராய்ச்சி மருத்துவமனை ,

Researchers speed identification of DNA regions that regulate gene expression


Date Time
Researchers speed identification of DNA regions that regulate gene expression
Corresponding author Yong Cheng, Ph.D., of the St. Jude Departments of Hematology and Computational Biology, helped develop a highly efficient method in identifying the genetic switches that regulate gene expression.
St. Jude Children’s Research Hospital scientists have developed an integrated, high-throughput system to better understand and possibly manipulate gene expression for treatment of disorders such as sickle cell disease and beta thalassemia. The research appears today in the journal Nature Genetics.
Researchers used the system to identify dozens of DNA regulatory elements that act together to orchestrate the switch from fetal to adult hemoglobin expression. The method can also be used to study other diseases that involve gene regulation. ....

Peng Xu , Jingjing Chen , Qian Qi , Lance Palmer , Ruiqiong Wu , Akshay Sharma , Ross Hardison , Ruopeng Feng , Jingjing Zhang , Li Cheng , Tiffany Yee , Yichao Li , Mitchell Weiss , Jude Children Research Hospital , National Institutes Of Health , Pennsylvania State University , Jude Department Of Hematology , Computational Biology , Jude Children , Research Hospital , Jude Departments , Jude Sickle Cell Genome Project , Pennsylvania State , National Institutes , பெங் ஐயூ , லான்ஸ் பாமர் ,

Can Public Entrepreneurship Solve Government's Complex Problems?


Can Public Entrepreneurship Solve Government’s Complex Problems?
A new book by a founding member of Boston’s Urban Mechanics calls for changing how government addresses its problems and provides an operating manual for generating new ideas and putting them into practice.
Dustin Haisler, Futures and Innovation Editor
  |  
January 22, 2021
  |  Opinion
There were no shortages of complex challenges that public-sector leaders had to navigate in 2020, from a global pandemic to the collateral fiscal constraints that followed. These problems forced agencies to respond rapidly in a new, unfamiliar landscape. After the first wave of the pandemic, the common phrase heard from government leaders was that years’ worth of work and progress had been completed in a matter of months. ....

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