Rocket Pharmaceuticals is conducting a clinical trial using a gene therapy for infantile malignant osteopetrosis (IMO), a rare and life-threatening disorder that develops in infancy. IMO is caused by defective bone cell…
Plasma is a component of blood that carries proteins called antibodies that are usually involved in defending our bodies against viral infections. Blood plasma from patients that have recovered from COVID-19,…
Acute respiratory distress syndrome (ARDS) is a life-threatening lung injury that occurs when fluid leaks into the lungs. As a result, breathing becomes difficult and oxygen cannot get into the…
Sickle Cell Disease (SCD) is an inherited blood disorder caused by a single gene mutation that results in the production of "sickle" shaped red blood cells. It affects an estimated…
Retinitis pigmentosa is a blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing…
Retinitis pigmentosa is blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing cells…
Parkinson's Disease is a neurodegenerative disease that is thought to be the outcome of the gradual breakdown of dopaminergic neurons in the brain, which are a type of cell that…
Limbal stem cell deficiency (LSCD) is a blinding corneal disease. LSCD is caused by a decrease in the number and/or function of limbal stem cells (LSCs), a type of stem cell…
Cystinosis is a rare disease that primarily affects children and young adults, and leads to premature death, usually in early adulthood. Patients inherit defective copies of a gene called CTNS,…
Breast cancer is the second-most common cancer in women, both in the United States (US) and worldwide. Some types of breast cancer have a high likelihood of metastasizing to the…
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