Diseases, Conditions, Syndromes

Toward a reliable oral treatment for sickle cell disease

For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...

Genetics

Cellular benefits of gene therapy seen decades after treatment

An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted ...

Medical research

How bone marrow regenerates after chemotherapy

Chemotherapy has a damaging effect on hematopoietic stem and progenitor cells (HSPCs) in bone marrow. However, once chemotherapy ends, HSPCs regenerate, a process that has remained unknown—until now. In a new study, researchers ...

Genetics

Induced pluripotent stem cells reveal causes of disease

Induced pluripotent stem cells (iPSC) are suitable for discovering the genes that underly complex, rare genetic diseases. Scientists from the German Cancer Research Center (DKFZ) and the European Molecular Biology Laboratory ...

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