摘要
Advances in sequencing and molecular technology now allow us to understand the genetic underpinnings of complex diseases such as neurological disorders.Genetic variations(or mutations) in the DNA sequence of single genes have been implicated in neurological diseases such as Huntington's disease and spinal muscular atrophy.As a result,the development of gene therapies for neurological diseases is now a feasible endeavor.
基金
funded by grants from the National Health and Medical Research Council of Australia (1185600
to GSL)
Retina Australia (to GSL)
The Center for Eye Research Australia receives Operational Infrastructure Support from the Victorian Government。
