Gene therapy in inherited blood disorders: A spotlight on sickle cell disease and beta‑thalassaemia

  Inherited blood disorders are a group of conditions that alter the production, structure or function of blood cells, often requiring coordinated long-term management of patients. Haemoglobinopathies represent a subgroup of inherited blood disorders characterised by genetic changes in the haemoglobin, a key protein in the blood responsible for oxygen transport. Sickle cell disease (SCD) and beta-thalassaemia are two …

Parkinson’s disease: A hopeful future

  Parkinson’s disease is the fastest growing neurological condition in the world. Between 1990 and 2015, the number of people with this disease doubled to over 6 million [1]. This number is predicted to double again to 12 million by 2040, primarily because of an ageing population [1]. In the UK, the lifetime risk of …

Epilepsy Awareness: A spotlight on Dravet Syndrome

  Background Epilepsy is a chronic neurological condition that affects approximately 50 million people worldwide. It is characterised by recurrent seizures due to excessive electrical discharges in the brain [1]. Seizures experienced by individuals with epilepsy can be categorised as generalised (affecting both sides of the brain) or focal (located in one specific area of the …

Jeans for Genes Week 2021: Spotlight on cystic fibrosis

  Jeans for Genes is an annual campaign that aims to raise awareness of the challenges faced by people living with a genetic disorder. The campaign encourages children and adults to wear jeans to school and the workplace in exchange for a small donation. The funds raised are distributed as grants to disorder-specific charities and …