Macrophage colony-stimulating factor 1 receptor (CSF-1R) is a transmembrane tyrosine kinase receptor expressed in brain microglia, and mutations in the CSF1R gene have been linked to adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).
Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
The FDA has cleared Prime Medicine Inc.’s IND application for PM-359 for the treatment of chronic granulomatous disease (CGD), enabling initiation of a phase I/II trial in the U.S.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
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