Thalassemia News and Research

Latest Thalassemia News and Research

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

First ever American gene-editing treatment using CRISPR for genetic disease

In a pathbreaking experiment, a 34-year-old mother of four from Mississippi is in the limelight for volunteering to be the subject of CRISPR-mediated gene editing in the hope of achieving a cure for the painful sickle cell anemia with which she has suffered all her life.

30 Jul 2019

Better education and communication critical for increasing blood donation among minorities

Better community education and communication are critical for increasing levels of blood donation among minorities, according to a study by researchers at Georgia State University and Georgia Southern University.

14 Jun 2019

More screening and new treatments needed for sickle cell disease

The promise of new treatments for sickle cell disease, a group of inherited red blood cell disorders, has never been so great, but it will only be realized if there is constant investment in health policies such as programs for screening newborns, especially in sub-Saharan Africa, where most cases occur.

13 Jun 2019

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

Thalassemia News and Research

Latest Thalassemia News and Research

Researchers make breakthrough in gene therapy for sickle cell disease

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

First ever American gene-editing treatment using CRISPR for genetic disease

Better education and communication critical for increasing blood donation among minorities

More screening and new treatments needed for sickle cell disease

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

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

Thalassemia News and Research

Latest Thalassemia News and Research

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

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