Gene therapy has been successful in treating angiodema, a genetic disorder that causes painful swelling attacks.
The single-dose gene therapy using Crispr-Cas9 showed potential as a permanent cure for angiodema and has the potential to be a treatment for other genetic conditions.
Argentinian couple moves to US to allow their toddler to join gene therapy trial
Parents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.
Gene therapy has been successful in treating angiodema, a genetic disorder that causes painful swelling attacks.
The single-dose gene therapy using Crispr-Cas9 showed potential as a permanent cure for angiodema and has the potential to be a treatment for other genetic conditions.
Gene-Based Therapy Restores Cellular Development and Function in Brain Cells From People With Timothy Syndrome
A potential new therapy for Timothy syndrome shows promise in restoring typical cellular function in 3D structures created from patients' cells.
Antisense oligonucleotide therapeutic approach for Timothy syndrome - Nature
Timothy syndrome type 1 (TS1) is a severe genetic disorder affecting multiple organ systems and associated with neuropsychiatric conditions due to a pathogenic variant in CACNA1C.
Researchers Expand Understanding of Genetic Mechanisms Underlying Fragile X Syndrome
Fragile X syndrome (FXS) is a genetic disorder caused by changes to the FMR1 gene.
FXS involves extensive silencing of genes that play key roles in building tissues and making the brain function properly.
Scientists uncover genetic disorder that may affect thousands around world
Discovery of a genetic disorder with severe disabilities affecting hundreds of thousands worldwide.