A Teenager ’s Breakthrough Gene Therapy for Sickle Cell Disease

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) Hulu.com The Weekly (TV Program) Source Type: news

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Thalassemia major (TM) and sickle cell anemia (SCA) are the two most widespread inborn hemoglobinopathies worldwide. TM was the first congenital hemoglobinopathy to be cured by allogenic hematopoietic stem cell transplantation (HSCT),1 and SCA rapidly followed.2,3 Other than gene therapy,4 HSCT remains the only available curative treatment for these two diseases. Provided that an HLA-identical sibling donor is available, it should be performed as soon as possible for TM and in case of complications for SCA.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
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
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 https://www.fightaging.org/archives/2019/11/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
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
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
Abstract Sickle cell disease afflicts millions of people worldwide and approximately 100,000 Americans. Complications are myriad and arise as a result of complex pathological pathways 'downstream' to a point mutation in DNA, and include red blood cell membrane damage, inflammation, chronic hemolytic anemia with episodic vaso-occlusion, ischemia and pain, and ultimately risk of cumulative organ damage with reduced patient lifespan. The National Heart, Lung, and Blood Institute's 2014 evidence-based guideline for sickle cell disease -management states additional research is needed before investigational curative the...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
This article is protected by copyright. All rights reserved. PMID: 31231794 [PubMed - as supplied by publisher]
Source: Clinical Genetics - Category: Genetics & Stem Cells Authors: Tags: Clin Genet Source Type: research
A cutting-edge clinical trial made a breakthrough in its effort to cure sickle cell anemia. The painful genetic disease affects about 100,000 Americans every year, mostly African Americans. On "60 Minutes," Dr. Jon LaPook followed Jenelle Stephenson for more than a year as she underwent an innovative kind of gene therapy at the National Institutes of Health. The therapy uses HIV to treat patients, but the virus is weakened, so it cannot cause AIDS. LaPook, who watched his report Sunday night with four sickle cell anemia patients, and Stephenson join "CBS This Morning" to discuss the remarkable trial results.
Source: Health News: CBSNews.com - Category: Consumer Health News Source Type: news
A new gene therapy for sickle cell anemia has shown promise in early clinical trials after reversing the condition's symptoms in two adults, researchers report.
Source: Health News - UPI.com - Category: Consumer Health News Source Type: news
TUESDAY, Dec. 4, 2018 -- A new gene therapy shows early promise against sickle cell anemia, researchers say. The therapy targets the genetic flaw that causes sickle cell. In a small group of patients, researchers said the therapy appears safe and...
Source: Drugs.com - Daily MedNews - Category: General Medicine Source Type: news
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