Thalassemia News and Research

Latest Thalassemia News and Research

UNSW research improves fundamental understanding of the mechanism behind sickle cell disease

UNSW researchers have used CRISPR gene editing – a type of 'molecular scissors' – to understand how deletions in one area of the genome can affect the expression of nearby genes.

8 Apr 2022

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age.

22 Feb 2022

Gene therapy may be a curative treatment option for patients with transfusion-dependent beta-thalassemia

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine.

3 Feb 2022

Hydroxyurea therapy can improve or reverse cardiac complications in sickle cell anemia patients

Heart abnormalities are common complications of sickle cell anemia (SCA) that can contribute to severe disease symptoms and even death.

16 Sep 2021

Iron chelating properties of eltrombopag in pediatric acute myeloid leukemia

Oncotarget published "Eltrombopag and its iron chelating properties in pediatric acute myeloid leukemia" which reported that the standard treatment for pediatric AML currently consists in a combination of cytarabine and antracycline.

9 Aug 2021

A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests

An experimental drug reported in Nature Communications suggests that a "path is clearly achievable" to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations.

19 Jul 2021

Study examines safety of COVID-19 vaccination during lactation

A new preprint, recently released on the medRxiv* server, reports from real life in a cohort of mother-child dyads in Singapore. The mothers were lactating after having received COVID-19 vaccination.

25 Jun 2021

UCSF launches new initiative to propel the development of living therapeutics

UC San Francisco is launching a new initiative to propel the development of living therapeutics - a category of treatments broadly defined as human and microbial living cells that are selected, modified, or engineered to treat or cure disease - and bring them quickly to patients.

14 Jun 2021

Researchers develop proof-of-concept treatment for blood disorders

Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin.

1 Jun 2021

High-throughput system can regulate gene expression for treating sickle cell disease, beta thalassemia

St. Jude Children's Research Hospital scientists have developed an integrated, high-throughput system to better understand and possibly manipulate gene expression for treatment of disorders such as sickle cell disease and beta thalassemia. The research appears today in the journal Nature Genetics.

7 May 2021

Toward a transformative therapy for sickle cell patients

For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms.

9 Apr 2021

PNA-based gene editing technique can advance gene discovery

In an article published in the April 8 issue of Nature, the National Institutes of Health's Somatic Cell Gene Editing Consortium provided a detailed update on the progress of their nationwide effort to develop safer and more effective methods to edit the genomes of disease-relevant somatic cells and reduce the burden of disease caused by genetic changes.

8 Apr 2021

Research provides a new cornerstone of malaria's evolution with humans

New bioarchaeological research shows malaria has threatened human communities for more than 7000 years, earlier than when the onset of farming was thought to have sparked its devastating arrival.

13 Mar 2021

Combined liver-cytokine humanization enhances human red blood cell production and survival

In a new study by the Yale Department of Immunobiology and Yale Cancer Center, researchers report combined liver and growth factor humanization enhances human red blood cell production and survival in circulation the immunodeficient murine host.

5 Mar 2021

Study: TALEN is five times more efficient than CRISPR-Cas9 in tightly packed DNA

Researchers used single-molecule imaging to compare the genome-editing tools CRISPR-Cas9 and TALEN.

27 Jan 2021

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

Thanks to major progress in the understanding and management of rare congenital diseases and syndromes, many patients with these rare disorders are now living longer lives.

13 Jan 2021

Researchers develop telomerase gene therapy for treating different pathologies

After several years of research and many joint scientific publications, molecular biologists Maria Blasco (National Cancer Research Centre, CNIO) and Fàtima Bosch (Universitat Autònoma de Barcelona, UAB) have developed a telomerase gene therapy for the treatment of different pathologies related to shortening telomeres and, therefore, ageing.

2 Dec 2020

Majority of U.S. COVID-19 fatalities had underlying medical conditions

Early on in the coronavirus disease (COVID-19) pandemic, there was little data on the virus and how it affects the body. As the virus spread across the globe, data showed that some people were at a higher risk of developing severe disease and dying from the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection.

31 Aug 2020

Umbilical cord blood safely and effectively treats children with rare genetic disorders

Researchers at UPMC Children's Hospital of Pittsburgh found that infusing umbilical cord blood - a readily available source of stem cells - safely and effectively treated 44 children born with various non-cancerous genetic disorders, including sickle cell, thalassemia, Hunter syndrome, Krabbe disease, metachromatic leukodystrophy (MLD) and an array of immune deficiencies.

14 Jul 2020

Impact of COVID-19 on pregnancy and breastfed infants

Pregnant women are among those at high risk of developing COVID-19, as confirmed in Wuhan early in the pandemic. A new study from Portugal published on the preprint server medRxiv in May 2020 analyses the data on the impact of COVID-19 on pregnancy and disease outcomes, using published cases of pregnant women diagnosed with the illness.

4 May 2020

Thalassemia News and Research

Latest Thalassemia News and Research

UNSW research improves fundamental understanding of the mechanism behind sickle cell disease

Gene therapy leads to durable transfusion independence in youngest patients with thalassemia

Gene therapy may be a curative treatment option for patients with transfusion-dependent beta-thalassemia

Hydroxyurea therapy can improve or reverse cardiac complications in sickle cell anemia patients

Iron chelating properties of eltrombopag in pediatric acute myeloid leukemia

A path to treat currently untreatable cases of cystic fibrosis is clearly achievable, study suggests

Study examines safety of COVID-19 vaccination during lactation

UCSF launches new initiative to propel the development of living therapeutics

Researchers develop proof-of-concept treatment for blood disorders

High-throughput system can regulate gene expression for treating sickle cell disease, beta thalassemia

Toward a transformative therapy for sickle cell patients

PNA-based gene editing technique can advance gene discovery

Research provides a new cornerstone of malaria's evolution with humans

Combined liver-cytokine humanization enhances human red blood cell production and survival

Study: TALEN is five times more efficient than CRISPR-Cas9 in tightly packed DNA

New taxonomic classification of non-skeletal rare congenital disorders with impact on bone physiology

Researchers develop telomerase gene therapy for treating different pathologies

Majority of U.S. COVID-19 fatalities had underlying medical conditions

Umbilical cord blood safely and effectively treats children with rare genetic disorders

Impact of COVID-19 on pregnancy and breastfed infants

One Health, One Mission: The World Organisation for Animal Health on the AMR Frontline

Uniting Against Resistance: The World Health Organization for World Antimicrobial Awareness Week 2023

Tackling Antimicrobial Resistance: A Conversation with Dr. Beverley Isherwood

Thalassemia News and Research

Latest Thalassemia News and Research

One Health, One Mission: The World Organisation for Animal Health on the AMR Frontline

Uniting Against Resistance: The World Health Organization for World Antimicrobial Awareness Week 2023

Tackling Antimicrobial Resistance: A Conversation with Dr. Beverley Isherwood

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