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A potentially safer, more effective gene therapy vector for blood disorders

 E-Mail Philadelphia, January 29, 2021 Researchers at Children s Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently used in gene therapy trials for those conditions. The vector, an engineered vehicle for delivering functional copies of the hemoglobin gene to correct a genetic abnormality, leads to the production of more hemoglobin with a lower dose, minimizing the risk of toxic side effects. The findings were published today in Molecular Therapy. These results have many potential benefits for the successful treatment of patients affected by beta-globinopathies like sickle cell disease and beta-thalassemia, including a better dose response, a minimized chance of clonal expansion and tumorigenesis, a reduced cost of therapy, and a potentially reduced need for chemotherapy or radiation before beginning gene therapy, said Laura Bre

CHOP Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders

CHOP Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders New preclinical study shows vector results in significantly more hemoglobin production than vectors currently used in gene therapy for sickle cell disease and beta-thalassemia News provided by Share this article Share this article PHILADELPHIA, Jan. 29, 2021 /PRNewswire/  Researchers at Children s Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently used in gene therapy trials for those conditions. The vector, an engineered vehicle for delivering functional copies of the hemoglobin gene to correct a genetic abnormality, leads to the production of more hemoglobin with a lower dose, minimizing the risk of toxic side effects.

Namoo, Tinker, Prison X & More Premiere at Sundance Film Festival This Week

This year there’s a scaled-down selection of titles to view, with Baobab Studios’ Namoo is Korean for “tree”, taking viewers on a journey through a man’s life with each branch a different memory. Created using Oculus’  Quill,the project will be coming to Oculus platforms later this year. And then there’s Prison X, Chapter 1: The Devil and The Sun, a new VR series from Quechua filmmaker Violeta Ayala. The first episode takes you into Bolivia’s infamous San Sebastian Prison as Inti, a young man imprisoned after his first job as a drug mule. “It was my world but it wasn’t a world you could capture with a camera,” says Ayala who grew up three blocks from the prison. “And I needed technology that wasn’t yet invented – virtual reality.”

There s more than 1 way to send a spacecraft to Venus

There’s more than 1 way to send a spacecraft to Venus A look at Rocket Lab’s private mission to Earth’s twin, and how NASA decides what planets to visit Updated:  Tags:  NASA last sent a spacecraft to orbit Earth’s nearest neighbor in 1990. Russia, formerly the Soviet Union, sent a whole slew of mostly successful missions to Venus, and Japan has an orbiter there now. The tricky part is sending a robotic mission to the surface. Most have only survived for a few hours. So why send a robot to a planet that will destroy it? The short answer: It can likely tell us more about our home planet.

Detailed text transcripts for TV channel - MSNBC - 20130609:19:54:00

we have esther arms, bob franklin, robert costa. we left off with you, robert. we have 77 cents on the dollar in 2012. does this affect those of color more than those who are not? it is really distressing if those are the statistics. this was past in 1963, part of jfk s new frontier program. 50 years on, it is clear there are some problems remaining in the work place. you think congress is paying attention. you hope they are. i think it is something we all should be paying attention to as the journalists. what is are headline? the new frontier at 50, equal pay act continues 50 years later but some problems remain. bob? well, i m sort of stuck on the 77% pay gap there. i am sort of a 23% empty kind of guy. my headline would be, equal pay act at 50, still hollow after all these years.

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