First-in-human universal gene therapy for blood disorder

Paper coming out in Cell Stem Cell 11/11

2024-11-11

(Press-News.org) Diamond-Blackfan anemia (DBA) is potentially life-threatening condition that can cause severe anemia and other abnormalities. It’s a rare genetic blood disorder that occurs when the bone marrow fails to make red blood cells. Currently the only cure is a stem cell transplant, but this is only a viable option for select patients. Otherwise, children with DBA require lifelong follow-up care to manage symptoms, such as steroids and blood transfusions.

New data publishing 11/11 in Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial for DBA through regulated GATA1 expression. Notably, it’s one of the first examples of treating 30 different genetic mutations with a single vector. This clinical-grade vector is ready, and the team hopes to take it to clinical trial next.Vijay Sankaran, MD, PhD, lead and corresponding author of the paper, is available for comment if you’re interested in learning more.

Please reach out to Joelle Zaslow (joelle.zaslow@childrens.harvard.edu), Research and Science Communications Manager at Boston Children's Hospital, with any questions or media inquiries.

END

ELSE PRESS RELEASES FROM THIS DATE:

Study: Older adult prostate cancer patients are increasingly being overtreated

2024-11-11

Increasing percentages of some older U.S. men with intermediate-risk and high-risk prostate cancers are undergoing treatments that carry risks of side effects that can significantly reduce the quality of life without extending life, according to a new study led by Cedars-Sinai. This trend is problematic because these men may not have life expectancies that would allow them to receive the benefits of more aggressive treatments. The research findings were published in the peer-reviewed journal JAMA Internal Medicine. Prostate cancer is the second-most common cancer in the U.S., exceeded ...

Experiences of discrimination linked to postpartum weight retention

2024-11-11

PITTSBURGH, November 11, 2024 – Researchers have been unable to explain why after giving birth, Black patients are two to three times as likely to retain or gain additional weight compared to their white counterparts, even when pre-pregnancy weight and gestational-weight trajectories are comparable. A first-of-its-kind study by University of Pittsburgh epidemiologists points to the stress of lived experiences with racism and gender-based discrimination as a possible explanation. The study was reported today in the American Journal of Epidemiology. Since postpartum weight retention is associated with increased cardiovascular risk and other negative health outcomes that persist ...

New python package, ERTool, developed for efficient multi-source evidence fusion

2024-11-11

Researchers from Peking University have developed ERTool, an open-source Python package designed to simplify the implementation of the Evidential Reasoning (ER) approach for multi-source evidence fusion. This tool addresses the challenges of integrating data from multiple sources in uncertain decision-making environments. The results are published in Health Data Science. Multi-source evidence fusion plays a critical role in fields such as healthcare management, business analytics, and environmental risk assessment. However, the traditional application of the ER approach has been complicated, requiring expertise in coding. To overcome these challenges, Associate Research Professor Guilan ...

AI-based software 'guide' childbirth by ‘seeing’ the baby’s position in real time

2024-11-11

A new AI-based software has been developed that can be integrated with an ultrasound device to ‘guide’ childbirth by providing precise, real-time information on the baby’s head position. It can clearly indicate to operators—using a traffic light system—whether to proceed with a natural descent in the birth canal, whether to use a vacuum extractor, or even if an emergency cesarean is needed. This tool, which could be available in delivery rooms starting in 2028, was developed and validated as part of a ...

Arab scholars from around the globe convene in Qatar to advance science and research

2024-11-11

Doha, November 11, 2024 – The Arab Global Scholars (AGS) community gathered at Hamad Bin Khalifa University (HBKU) in Qatar Foundation’s Education City last week to herald the latest evolution of an initiative nurturing a contemporary renaissance of Arab science and research. An initiative led by Qatar Foundation, AGS aims to reconnect scholars and intellectuals with roots in the Arab world back to their region to contribute to its positive development and long-term future. Its beginnings going back almost two decades, AGS has solidified itself as an innovation-focused community linking 895 Arab scholars, as well as research ...

Gender inequality ingrained in global climate negotiations, say researchers

2024-11-11

Climate governance is dominated by men, yet the health impacts of the climate crisis often affect women, girls, and gender-diverse people disproportionately, argue researchers ahead of the upcoming 29th United Nations Climate Summit (COP29) in Azerbaijan. In an article published today in Lancet Planetary Health, a team of researchers – including several from the University of Cambridge – argue that much more needs to be done to mitigate the impacts of climate change on women, girls and gender-diverse individuals. Focusing specifically on the intersection between climate change, gender, and human health, ...

There's no time like the present for integrating climate and violence policies: The opportunity is right

2024-11-11

The dual goals of climate action and ending violence against children can be achieved according to a new paper by Columbia University Mailman School of Public Health and colleagues at the London School of Hygiene and Tropical Medicine. For the first time, governments are committing funds and making concrete pledges to reach the UN Sustainable Development Goal target of ending violence against children. On November 7th and 8th, the Government of Colombia, with support of the Government of Sweden, UNICEF, the UN Special Representative of the Secretary-General on Violence Against Children, and the World Health Organization will attend the first Global Ministerial Conference on ending ...

Breaking free from negative thoughts using a scientifically gamified app

2024-11-11

(Toronto, November 11, 2024) A new study published in the Journal of Medical Internet Research by JMIR Publications reveals promising results from a digital health intervention that is based on a decade of research at Harvard Medical School and designed to alleviate depressive symptoms. The study, titled "Facilitating Thought Progression to Reduce Depressive Symptoms: Randomized Controlled Trial," found that participants experienced substantial reduction of depressive symptoms by using a gamified mobile app focused on disrupting ruminative thinking. Led by Prof. Moshe Bar and colleagues, the research ...

The emerging role of (p)ppGpp in DNA repair and associated bacterial survival against fluoroquinolones

2024-11-11

Bacteria frequently encounter adverse environmental conditions, such as nutrient scarcity and antibiotic exposure, which can induce DNA damage. Efficient DNA repair mechanisms are essential for bacterial survival, particularly under such stress conditions. A critical player in these processes is the signaling molecule (p)ppGpp, a phosphorylated guanosine synthesized by bacteria during periods of stress. Initially discovered in Escherichia coli under amino acid starvation, (p)ppGpp is now recognized for its broader roles in modulating cellular functions essential for DNA repair and stress response. By regulating diverse cellular processes, (p)ppGpp not ...

People with fewer resources seen as less trustworthy across cultures, new research shows

2024-11-11

New research in Social Psychological and Personality Science identifies a widespread stereotype linking wealth to perceived trustworthiness across diverse cultures. The research, led by Mélusine Boon-Falleur from the Center for Research on Social Inequalities at Sciences Po in Paris, shows that individuals with fewer material resources are consistently viewed as less trustworthy. The study, conducted across eight countries including Brazil, Colombia, Democratic Republic of Congo, India, France, Nigeria, Philippines, and the United Kingdom, employed a novel method to uncover stereotypes while avoiding social desirability bias. "People ...

Aligning Science Across Parkinson’s (ASAP) launches a second funding opportunity to accelerate novel tool development to advance Parkinson's disease research

First-in-human universal gene therapy for blood disorder

ELSE PRESS RELEASES FROM THIS DATE:

LAST 30 PRESS RELEASES: