Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

Transposable Elements Drive a Critical Transition in Early Embryogenesis

Researchers at Sinai Health have discovered that DNA elements known as transposons, which may travel throughout the genome, control a crucial stage in early human development instead of human genes.

16 Oct 2024

Transposable Elements Are Key to Normal Progression of Embryonic Cells

A critical transition in early human development is regulated not by our own genes, but by DNA elements called transposons that can move around the genome, Sinai Health researchers have found.

16 Oct 2024

Retrotransposon-Based PRINT Technique Holds Potential for Precise Gene Insertion

The recent approval of a CRISPR-Cas9 therapy for sickle cell disease demonstrates that gene editing tools can do a superb job knocking out genes to cure hereditary disease.

22 Feb 2024

Study Analyzes the Development of a Novel Non-Viral Gene Therapy Procedure

The research group of Professor Kamimura in Niigata University have applied the novel, liver lobe-specific hydrodynamic delivery procedure to primates (baboons) for the first time.

9 Jun 2023

Tardigrade proteins for significant advancement in human health

Researchers at the University of Wyoming have made a significant discovery that could one day allow people living in areas without refrigeration to access life-saving treatments.

21 Mar 2023

New puzzle challenge taps into the intelligence of Eterna’s users to advance RNA therapeutics

Researchers at UC Santa Cruz working to develop novel RNA-based medicines are teaming up with a new group of collaborators-;players of the online game Eterna.

22 Feb 2023

Newly developed tool helps snip out faulty genes in a safe manner

Scientists from the Massachusetts Institute of Technology (MIT) have come up with a new tool that has the potential to detach faulty genes and exchange them with new ones in a highly safe and effective way.

From Massachusetts Institute of Technology (MIT) 25 Nov 2022

Immunological mechanisms behind the development of alloantibodies

Hemophilia A is the most frequent severe variant of hemophilia. It nearly mostly affects men. The condition is frequently treatable, although not for all patients. A study at the University of Bonn has now highlighted a critical mechanism required for the therapy to be efficient.

28 Oct 2022

Study provides insights into the biological mechanisms behind the severe form of COVID-19

A total of 16 novel genetic variations linked to severe Covid-19 have been discovered, including ones that affect blood coagulation, immunological response, and inflammation severity.

8 Mar 2022

Key discovery could prevent treatment failure in people with severe hemophilia A

Recently, researchers achieved a crucial discovery that could mitigate and eliminate immune responses causing treatment failure in people with hemophilia A.

20 Apr 2021

Lipid-based nanoparticles can help treat genetic disorders

Introducing genetic material into the body to treat diseases can work, but making sure that those materials are safely sent to the right location is complicated.

24 Aug 2020

Shielded stem cells can improve heart healing

Ghanta, R.K., et al. (2020) Immune-modulatory alginate protects mesenchymal stem cells for sustained delivery of reparative factors to ischemic myocardium. Biomaterials Science. doi.org/10.1039/D0BM00855A.

19 Aug 2020

A new cell-based gene therapy approach to correct hemophilia A

In a new study from the Wake Forest Institute for Regenerative Medicine (WFIRM) researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.

29 Jul 2020

New strategy can help treat diabetes and other diseases

Transplanted islet cells that release insulin during very low blood sugar levels offer a potential way for treating diabetes.

From Massachusetts Institute of Technology (MIT) 31 Mar 2020

Intact Protein Mass Spectrometry of Cell Culture Harvest Guides Cell Line Development for Trispecific Antibodies

A team in Massachusetts has established a method of profiling of mispairing in antibody assembly.

18 Feb 2020