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A step closer to gene therapy that restores hearing for congenitally deaf

A step closer to gene therapy that restores hearing for congenitally deaf

Findings from a new study have brought the possibility of gene therapy that could restore hearing in the congenitally deaf one step closer. Mutations in...
Hope for rare disease patients: an approach to funding gene therapy

Hope for rare disease patients: an approach to funding gene therapy

Roger Johansson, Vice President and General Manager – Northern Europe with the global biopharmaceutical company PTC Therapeutics, discusses the need to establish an environment...
Combining machine learning and cell engineering for cancer therapies

Combining machine learning and cell engineering for cancer therapies

Scientists are tackling the ‘Holy Grail’ of oncology by combing machine learning and cell engineering to create ‘living medicines’ that precisely target cancer tumours. Scientists...
Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency

Non-hereditary mutation acts as natural gene therapy for GATA2 deficiency

Scientists have identified a non-inherited mutation in blood cells from a patient with GATA2 deficiency that may have prevented bone marrow failure and other...
Tumour-tropic liposome technology could pave way for cancer treatments

Tumour-tropic liposome technology could pave way for cancer treatments

A collaboration between Toshiba and Shinshu University has resulted in the development of a ‘tumour-tropic liposome technology’ for gene therapy that could pave the...
Mitchell disease: solving the medical mystery

Mitchell disease: solving the medical mystery

Scientists have uncovered a genetic mutation that links to the rare Mitchell disease. A team of researchers led by Dr Hugo Bellen at Baylor College...
Gene therapy may hold key to treating life-threatening Danon disease

Gene therapy may hold key to treating life-threatening Danon disease

A new study has found that  gene therapy improved cardiac, muscle and liver function in Danon disease mouse models. Danon disease is a very rare,...
biobanking scientist putting a container for storage

edu-BRoTHER: an innovative student exchange programme providing biobank skills

University Regensburg Professor Christoph Brochhausen showcases the edu-BRoTHER biobanking student exchange project Biobanks are an increasingly significant resource in healthcare research. Professor Christoph Brochhausen, of...
woman with knee pain

NUI Galway launch €7.45m research project into osteoarthritis

The EU Horizon 2020 project will research the robotic production of groundbreaking regenerative stem cell therapies The €7.45m AutoCRAT project will enable researchers to develop...
Astronaut floating in space

Space, neurogenetics and nervous system research

Have you ever wondered about how space alters the mechanics of human muscles? Health Europa Quarterly spoke with Bertrand Fontaine, Director of the Institute...
medical graph

The dawn of gene therapy for rare diseases

Andrew Obenshain, Chief of Wings at biotechnology firm bluebird bio, tells HEQ about the future of gene therapies in the treatment of rare diseases Up...
doctor holding test tube in front of DNA strand

bluebird bio announces launch of beta thalassaemia gene therapy in Germany

Zynteglo is a one-time gene therapy that addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent. The one-time...
Mother Nature provides new gene therapy strategy to reverse disease

Mother Nature provides new gene therapy strategy to reverse disease

Scientists have developed a new gene therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that...
Ancient worm reveals how to destroy toxic cells in Parkinson's disease

Ancient worm reveals how to destroy toxic cells in Huntington’s disease

Insights from a new study found that microRNAs are important in controlling protein aggregates. In the study scientists have discovered that an ancient worm could...
Gene therapy trial offers hope for Sickle Cell Disease

Gene therapy trial offers hope for Sickle Cell Disease

Sickle Cell Disease is a serious and debilitating genetic disease caused by a mutation in the β-globin gene and there are currently no known...
Measuring stressed cells to understand the development of disease

Measuring stressed cells to understand the development of disease

When cells are stressed, they initiate a complex and precisely regulated response to prevent permanent damage. Researchers from Goethe University have developed a new protoeomics...
Artificial Intelligence has potential to transform gene therapy

Artificial Intelligence has potential to transform gene therapy

New research demonstrates the power of a comprehensive machine-guided approach to engineer improved capsids for gene therapy delivery. The research, Comprehensive AAV capsid fitness landscape...
Gene patent reform in the US and the knock-on effect in Europe

Gene patent reform in the US and the knock-on effect in Europe

Rosie McDowell, an attorney at EIP shares her thoughts on gene patent reform and the potential impact it might have across the globe. This year...
C difficile infection: The search for new Clostridioides difficile vaccine

C difficile infection: The search for new Clostridioides difficile vaccine

University of Exeter researchers have made a breakthrough in the quest for a new vaccine for killer hospital bug Clostridioides difficile (C difficile infection). The...

New viral vector for improved gene therapy in sickle cell disease created

Researchers at the National Institutes of Health (NIH) have developed a new and improved viral vector – a virus-based vehicle that delivers therapeutic genes...

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