Sentences with phrase «develop sickle cell»

Those individuals don't develop sickle cell disease.

Not exact matches

Emmaus Medical CEO Dr. Yutaka Niihara spent much of his career developing Endari, a recently approved FDA drug to treat sickle cell anemia.

Prior to insemination, a woman and man will both undergo genetic testing to determine whether or not their baby will develop a genetic disease like cystic fibrosis, sickle cell, or Huntington's disease.

Learn how sickle cell disease can affect your baby, if your baby could develop the condition too, and whether you shouldn't tak...

The genes for cf and sickle cell were not «developed».

A team of researchers at the Stanford University School of Medicine has used a gene - editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.

Harvard Stem Cell Institute (HSCI) scientists have taken the first steps toward developing a treatment that would make bone marrow — blood stem cell — transplantation safer and, as a result, more widely available to the millions of people living with blood disorders like sickle cell anemia, thalassemia, and ACell Institute (HSCI) scientists have taken the first steps toward developing a treatment that would make bone marrow — blood stem cell — transplantation safer and, as a result, more widely available to the millions of people living with blood disorders like sickle cell anemia, thalassemia, and Acell — transplantation safer and, as a result, more widely available to the millions of people living with blood disorders like sickle cell anemia, thalassemia, and Acell anemia, thalassemia, and AIDS.

For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and test a «curative therapy» for the first molecular disease: sickle cell disease.

The classic example is the sickle cell gene — people with one copy of the gene are strongly protected against malaria but those with two copies of the gene develop a life - threatening condition known as sickle - cell disease.

In a poster presentation (Abstract # 3379), Yunus Alapan, Umut Gurkan PhD and Jane Little, MD presented promising findings related to new technology aimed at facilitating early detection of sickle cell disease for infants in developing countries.

The National Heart, Lung, and Blood Institute (NHLBI) has released the first comprehensive, evidence - based guidelines for management of sickle cell disease from birth to end of life, based on recommendations developed by a nationwide team of experts co-chaired by a UT Southwestern Medical Center hematologist.

«Developing first comprehensive guidelines for management of sickle cell disease.»

Ortiz Genovese listed sickle cell disease, malaria and dengue among the diseases that collectively are responsible for hundreds of thousands of deaths per year in the developing world.

New preclinical research on the molecular mechanisms responsible for sickle cell disease could aid efforts to develop much needed treatments for this devastating blood disorder that affects millions worldwide.

«Sickle cell gene linked to elevated risk of developing kidney failure.»

Boston Children's Hospital has offered non-exclusive licenses to for - profit entities on a patent developed by Orkin's laboratory regarding BCL11A, a genetic switch regulating hemoglobin production that is expected to form the basis of clinical trials for gene therapy and gene editing for sickle cell disease and thalassemia.

«The one thing that I think that is useful is maybe when people write knockout papers they might describe the housing conditions in more detail,» says Chris Paszty, scientific director at the biotech company Amgen, Inc., headquartered in Thousand Oaks, Calif., who as a postdoctoral researcher at Lawrence Berkeley National Laboratory developed a mouse model for the study of sickle cell anemia.

«For example, despite matching the ABO blood group antigens during red blood cell (RBC) transfusion, up to 45 percent of chronically transfused patients, such as those with sickle cell disease or thalassemia, develop antibodies to mismatched minor antigens on transfused RBCs; this process is known as alloimmunization.»

This year's Action Award honorees include global leaders in the fight to end cancer as we know it; a world leader in advancing the emerging field of regenerative medicine and game - changing cell therapy medical treatments; the president of a non-profit group focused on developing cures for chronic, debilitating and fatal diseases; a sickle cell and stem cell advocate and founder / science administrator of the Axis Advocacy; and the founding director of the Institute for Integrated Cell - Material Sciences (iCeMS) at Japan's Kyoto Universcell therapy medical treatments; the president of a non-profit group focused on developing cures for chronic, debilitating and fatal diseases; a sickle cell and stem cell advocate and founder / science administrator of the Axis Advocacy; and the founding director of the Institute for Integrated Cell - Material Sciences (iCeMS) at Japan's Kyoto Universcell and stem cell advocate and founder / science administrator of the Axis Advocacy; and the founding director of the Institute for Integrated Cell - Material Sciences (iCeMS) at Japan's Kyoto Universcell advocate and founder / science administrator of the Axis Advocacy; and the founding director of the Institute for Integrated Cell - Material Sciences (iCeMS) at Japan's Kyoto UniversCell - Material Sciences (iCeMS) at Japan's Kyoto University.

CRISPR / Cas9 could be used to develop therapies for humans for genetic blood diseases such as sickle cell or thalassemia, and this paper does not change that potential.

As the stem cell field works toward developing treatments for RP, sickle cell and other diseases, the pursuit is strengthened and the process enhanced by the partnership of patients and disease advocates.

Scientists developing new cell therapy methods for the treatment of disorders such as sickle cell disease or leukemia need to consider how their culture conditions may affect the success of their translational research.

The long - term vision is to apply these technologies to other genetic diseases, such as sickle cell disease, and to explore applications in developing effective immune therapies for cancer.

In contrast to Mendelian disorders (e.g., Huntington's disease, sickle cell anemia) in which variation in a single gene causes disease, common complex disorders, such as heart disease, diabetes, and most cancers, develop as a result of both genetic and environmental factors.

Salk researchers reprogrammed skins cells taken from a sickle cell disease patient into induced pluripotent stem cells (iPSCs), immature cells capable of developing into any type of bodily tissue.

LA JOLLA, CA — Researchers at the Salk Institute for Biological Studies have developed a way to use patients» own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood hemoglobin.

Dana - Farber / Boston Children's is leading the way in developing new treatment options for sickle cell disease.