Thalassemia News and Research

Latest Thalassemia News and Research

Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

Scientists at Fred Hutchinson Cancer Research Center took a step toward making gene therapy more practical by simplifying the way gene-editing instructions are delivered to cells.

28 May 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

New gene therapy cures babies with fatal 'Bubble Boy' disease

A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease.

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

Study illuminates how hot spots of genetic variation evolved in the human genome

What makes one person different from one another, and how did these differences evolve? A study by University at Buffalo biologists is illuminating one aspect of this complicated question. The research examines hot spots of genetic variation within the human genome, examining the sections of our DNA that are most likely to differ significantly from one person to another.

20 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

Antibody-based treatment could improve cure rate for blood and immune disorders

An antibody-based treatment can gently and effectively eliminate diseased blood-forming stem cells in the bone marrow to prepare for the transplantation of healthy stem cells, according to a study in mice by researchers at the Stanford University School of Medicine.

12 Feb 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

Advances in gene therapy for inherited blood disorders

Gene therapy holds a lot of promise in medicine. If we could safely alter our own DNA, we might eliminate diseases our ancestors passed down to us.

4 Dec 2018

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Bluebird bio, Inc. and Gritstone Oncology, Inc. today announced a collaboration to research, develop and commercialize products for the treatment of cancer using cell therapy.

23 Aug 2018

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Regenacy Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company developing breakthrough treatments for diabetic and other peripheral neuropathies, today announced a collaboration with the Charcot-Marie-Tooth Association, a registered non-profit organization serving the hereditary neuropathy patient community, to validate the role of HDAC6 in multiple forms of Charcot-Marie-Tooth disease and evaluate the efficacy of ricolinostat, a selective HDAC6 inhibitor, in animal models.

2 Aug 2018

Therapy with L-Glutamine reduces pain in patients with sickle cell disease

UCSF Benioff Children's Hospital Oakland clinical researchers, in conjunction with other sickle cell centers and scientists at Emmaus Life Sciences, Inc., have demonstrated that therapy with L-Glutamine reduced the frequency of pain episodes in both pediatric and adult patients with sickle cell disease.

27 Jul 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

Study shows gene variations associated with malaria risk

Many people of African heritage are protected against malaria by inheriting a particular version of a gene, a large-scale study has shown.

25 Apr 2018

Smart Cells launches social campaign to support Rare Disease Day 2018

There are more than 6000 known rare diseases and over 30 million people living daily with them. 28th February marks Rare Disease Day 2018, a date that highlights awareness around these conditions and the need for more research into them.

From Smart Cells International Ltd 23 Feb 2018

Company-sponsored CRISPR clinical studies to begin in 2018

This year could be a defining one for CRISPR, the gene editing technique, which has been hailed as an important breakthrough in laboratory research.

10 Jan 2018

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Sangamo Therapeutics, Inc. and Pfizer Inc. today announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.

8 Jan 2018

Thalassemia News and Research

Latest Thalassemia News and Research

Scientists use gold nanoparticles to edit genes tied to HIV, inherited blood disorders

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

New gene therapy cures babies with fatal 'Bubble Boy' disease

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

Study illuminates how hot spots of genetic variation evolved in the human genome

Deadly inherited blood disorders cured with bone marrow transplant

Antibody-based treatment could improve cure rate for blood and immune disorders

App that could detect level of anemia by looking at fingernails

Advances in gene therapy for inherited blood disorders

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Regenacy Pharmaceuticals partners with CMTA to advance ricolinostat for treating hereditary neuropathy

Therapy with L-Glutamine reduces pain in patients with sickle cell disease

Researchers identify enzyme as potential new drug target for blood disorders

Mice study shows promise for treating genetic conditions during fetal development

Research project offers hope for pediatric patients with thalassemia

Celgene to share new and updated data around novel hematological therapies

Study shows gene variations associated with malaria risk

Smart Cells launches social campaign to support Rare Disease Day 2018

Company-sponsored CRISPR clinical studies to begin in 2018

Sangamo and Pfizer partner for development of new zinc finger protein gene therapy to treat ALS

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

Thalassemia News and Research

Latest Thalassemia News and Research

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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