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New therapy could revolutionise treatment for fatal Sanfilippo Syndrome

New therapy could revolutionise treatment for fatal Sanfilippo Syndrome

A new combination therapy could revolutionise treatment for children living with a rare and fatal lysosomal storage disorder called Sanfilippo Syndrome. GlycoNet researchers are using...
Is CRISPR gene therapy for rare Angelman Syndrome on the horizon?

Is CRISPR gene therapy for rare Angelman Syndrome on the horizon?

For the first time, scientists have shown how gene editing techniques could be used as therapy for the rare neurodevelopmental disorder – Angelman syndrome. Babies...
Successful treatment of patients with the autoimmune disease lupus

Successful treatment of patients with the autoimmune disease Lupus

A team of researchers have successfully treated two patients with the autoimmune disease systemic lupus erythematosus for the first time. The team from Charité -...
Understanding MPS and other similar Lysosomal diseases

Understanding MPS and other similar Lysosomal diseases

MPS Austria wants to make everyday life easier for children with Mucopolysaccharidosis and other similar Lysosomal diseases, and their families, by supporting them with...
Groundbreaking gene therapy could cure haemophilia

Groundbreaking gene therapy could cure haemophilia

A ‘cure’ for haemophilia is one step closer, following results published in the New England Journal of Medicine of a groundbreaking gene therapy trial...

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