Beta Thalassemia News and Research

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Beta-thalassemia (ß-thalassemia) is a form of thalassemia due to mutations in the HBB gene on chromosome 11 , inherited in an autosomal recessive fashion.

Pioneering CRISPR therapy leaves two patients free of blood disease symptoms

The first two clinical trials for CRISPR-based treatments for inherited blood disorders have been successful.

20 Nov 2019

Deterministic mechanoporation of cells en masse to cure cancer

What if you could cure cancer by re-engineering patients' cells to better target and destroy their own tumors? With the advent of powerful new cellular engineering technologies, this is no longer the stuff of science fiction.

13 Nov 2019

Carnegie Mellon and Yale win NIH grant to advance gene editing technique

A research team from Carnegie Mellon University and Yale University will advance their innovative, synthetic nucleic acid-based gene editing technique under a new grant from the National Institutes of Health's Somatic Cell Genome Editing Program.

7 Oct 2019

Researchers make breakthrough in gene therapy for sickle cell disease

Researchers at the National Institutes of Health have developed a new and improved viral vector--a virus-based vehicle that delivers therapeutic genes--for use in gene therapy for sickle cell disease.

2 Oct 2019

Genetic engineering could open possibilities for patients with Parkinson’s disease

People with Parkinson’s disease experience loss of dopamine-producing brain cells, leading to tremors, rigidity, bradykinesia, and postural instability, the primary symptoms of the disease.

11 Sep 2019

Study compares effectiveness and cost of gene therapy and HSCT in major beta-thalassemia

Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation

18 Apr 2019

Researchers develop new strategy to treat sickle cell disease and beta thalassemia

Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases -- sickle cell disease and beta thalassemia -- applying CRISPR-Cas9 gene editing to patients' own blood stem cells.

29 Mar 2019

Deadly inherited blood disorders cured with bone marrow transplant

Doubling the low amount of total body radiation delivered to patients undergoing bone marrow transplants with donor cells that are only "half-matched" increased the rate of engraftment from only about 50 percent to nearly 100 percent, according to a new study by Johns Hopkins researchers.

15 Mar 2019

App that could detect level of anemia by looking at fingernails

Researchers from the Emory University have developed a new phone app that needs just a colour photograph of the nail beds of an individual to detect anemia in them. The results of the report titled, “Smartphone app for non-invasive detection of anemia using only patient-sourced photos,” were published in the latest issue of the journal Nature Communications.

5 Dec 2018

Researchers identify enzyme as potential new drug target for blood disorders

Medical researchers have identified a key signaling protein that regulates hemoglobin production in red blood cells, offering a possible target for a future innovative drug to treat sickle cell disease.

20 Jul 2018

Mice study shows promise for treating genetic conditions during fetal development

Researchers at Carnegie Mellon University and Yale University have for the first time used a gene editing technique to successfully cure a genetic condition in a mouse model.

10 Jul 2018

Research project offers hope for pediatric patients with thalassemia

Children born with thalassaemia, a genetic blood disease, cannot create normal haemoglobin, which is needed to produce healthy red blood cells. Regular blood transfusions are the only treatment available, but iron overload is inevitable.

1 Jun 2018

Celgene to share new and updated data around novel hematological therapies

Celgene Corporation today announced that data from a broad range of early and late stage studies evaluating Celgene investigational agents and investigational uses of marketed products will be presented at the 23rd European Hematology Association annual meeting in Stockholm, Sweden, from June 14-17, 2018.

22 May 2018

“Chemical surgery” on embryos to free them of a gene causing thalassemia

A team of Chinese researchers have for the first time “mended” defective embryos using chemical surgery to free them of a faulty gene that leads to beta thalassemia. Until now this defect was found to be incorrigible caused due to a single misspelling in the DNA code. Beta thalassemia is a dreaded blood disorder wherein the child is unable to make healthy blood needing blood transfusions on a regular basis. The study was published this week in the journal Protein and Cell.

2 Oct 2017

Metabolism can impact host's susceptibility to develop malaria

Researchers from Instituto de Medicina Molecular Lisboa have found that the host's susceptibility to develop malaria depends on his or her metabolic state, which can be easily manipulated through external stimuli such as dietary patterns.

25 Sep 2017

Study provides insight into development of red blood cells

Every cell in the body, whether skin or muscle or brain, starts out as a generic cell that acquires its unique characteristics after undergoing a process of specialization. Nowhere is this process more dramatic than it is in red blood cells.

18 Aug 2017

US scientists make advances in modifying human embryos

American Scientists have managed to edit and improve the DNA of human embryos in an effort to correct the gene defects that cause inherited diseases.

27 Jul 2017

Innovative gene therapy allows complete remission of clinical signs of sickle cell disease

A team led by Pr. Marina Cavazzana conducted at Necker Children's Hospital, AP-HP and the Imagine Institute in October 2014 gene therapy in the context of a phase I clinical trial / II in a patient 13 years with severe sickle cell disease.

2 Mar 2017

Scientists find new way to use CRISPR gene editing to help fix sickle cell disease

An international team of scientists led by researchers at St. Jude Children's Research Hospital has found a way to use CRISPR gene editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients.

16 Aug 2016

Minihepcidins may potentially treat serious genetic blood diseases in children, adults

New research suggests that synthetic peptides called minihepcidins may potentially treat two serious genetic blood diseases in children and adults. Although those diseases, beta-thalassemia and polycythemia vera, have opposite effects on red blood cell production, treating animals with minihepcidin helps to restore normal levels of red blood cells and reduces spleen enlargement. It also controls the accumulation of excess levels of iron in beta-thalassemia that often causes severe toxic effects.

9 May 2016