Dominant inheritance means that on average 50% of the children of an affected person will be affected by the disorder. Another common feature of these disorders is that the causative genes are often characterised by mutational heterogeneity.
Therefore typically a large number of individual mutations in a single gene can lead to a particular disorder. For example any one of approximately 200 different mutations in the rhodopsin gene (RHO) can lead to adRP. Developing individual therapies for so many RHO mutations is practically impossible.Read More
Genable was acquired by Spark Technologies Inc; on March 7th 2016, for further information please visit www.sparktx.com. This transaction sees Genable become fully owned by Spark Therapeutics, a world leader in gene therapy development. RhoNova it’s product, which was acquired was based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries at Trinity College Dublin, Ireland