Gene Therapy May Aid In Sickle Cell Disease Treatment

Scientists report progress using gene therapy to treat sickle cell disease, a common and devastating genetic blood disorder.
Source: NPR Health and Science - Category: Consumer Health News Authors: Source Type: news

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Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure the disease, which afflicts 100,000 Americans.
Source: NYT Health - Category: Consumer Health News Authors: Tags: Sickle Cell Anemia Genetics and Heredity Genetic Engineering Stem Cells Clinical Trials Boston Children ' s Hospital Bluebird Bio Source Type: news
Doctors reset Helen Obando ’s DNA in an effort to cure her of a painful genetic blood disorder. She’s the youngest person to receive the treatment.
Source: NYT Health - Category: Consumer Health News Tags: Sickle Cell Anemia DNA (Deoxyribonucleic Acid) Genetics and Heredity Boston Children ' s Hospital Clinical Trials FX (TV Network) The Weekly (TV Program) Source Type: news
Authors: Howard J, Thein SL Abstract In countries with access to organized health care, survival of children with sickle cell disease (SCD) has greatly improved, resulting in a growing population of adults with SCD. Transition from pediatric to adult care presents many challenges for the patient, who now faces the reality of emerging complications in many organs that are cumulative, adding to other age-related nonsickle conditions that interact and add to the disease morbidity. We recommend regular comprehensive annual assessments, monitoring for early signs of organ damage and joint clinics with relevant specialis...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
Nature, Published online: 04 December 2019; doi:10.1038/d41586-019-03752-5Get rid of ‘ergodicity’ and a bunch of puzzling economic phenomena suddenly make sense. Plus, gene therapy tackles sickle-cell disease and doubts about whether tame animals necessarily get cuter.
Source: Nature AOP - Category: Research Authors: Source Type: research
Nature, Published online: 04 December 2019; doi:10.1038/d41586-019-03698-8After finally gaining traction as a potential treatment for certain genetic disorders, gene therapy tackles the challenge of sickle-cell disease.
Source: Nature AOP - Category: Research Authors: Source Type: research
This study demonstrates for the first time that senescent cells secrete functional LTs, significantly contributing to the LTs pool known to cause or exacerbate idiopathic pulmonary fibrosis. Against Senolytics There is no consensus in science that is so strong as to have no heretics. So here we have an interview with a naysayer on the matter of senolytic treatments, who argues that the loss of senescent cells in aged tissues will cause more harm to long-term health than the damage they will do by remaining. To be clear, I think this to be a ...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
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Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD) are the most common monogenic diseases worldwide, with a global carrier frequency of over 5%. With migration they are becoming more common worldwide, making their management and care an increasing concern for health care systems.BT is characterized by an imbalance in the α/β-globin chain ratio, ineffective erythropoiesis, chronic hemolytic anemia, and compensatory haemopoietic expansion. Globally, there are over 25,000 births each year with transfusion-dependent thalassemia (TDT). The current available tr...
Source: Mediterranean Journal of Hematology and Infectious Diseases - Category: Hematology Source Type: research
Some of the most cutting-edge — and effective — treatments in medicine are unaffordable to the majority of people who need them, with price tags sometimes exceeding $1 million. A new initiative from the National Institutes of Health (NIH) and the Bill &Melinda Gates Foundation is meant to change that unfortunate reality. Over the next four years, the NIH and the Gates Foundation will each invest $100 million toward developing gene-based cures for sickle cell disease and HIV, with a special focus on making these treatments available to the patients who need them most, the NIH announced Wednesday. Both HIV an...
Source: TIME: Health - Category: Consumer Health News Authors: Tags: Uncategorized onetime Research Source Type: news
This study reviews the current status of allo-HCT and autologous cellular therapies for SCD. Recent findings Alternative sources of allogeneic stem cells from unmatched donors such as cord blood and haploidentical donors are gaining traction. Early experience has shown that better conditioning regimens and graft-versus-host disease prophylaxis are needed before these donor sources can gain widespread use. Clinical trials are underway to determine the feasibility and efficacy of autologous transplantation with gene modified hematopoietic stem cells. Gene therapy strategies include HbS gene correction, gene addition, and ...
Source: Current Opinion in Hematology - Category: Hematology Tags: TRANSFUSION MEDICINE AND IMMUNOHEMATOLOGY: Edited by Steven L. Spitalnik Source Type: research
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