...-dependent toxicity. Summary: The preclinical gene therapy literature for treating RTT illustrates a duly diligent progression that begins with proof-of-concept studies and advances toward the development of safer, regulated MECP2 viral genome designs. This design progression was partly achieved through...

... or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements. Antisense oligonucleotide Gene...

.... Neurodevelopmental disorders CRISPR/dCas9 Gene therapy The correct functionality of the brain is ensured by the accomplishment of complex processes occurring in both embryonic and postnatal life and tightly regulated and coordinated by transcriptional programs [1, 2]. Those processes, which...

.... In recent years, there has been a rapid expansion of research into gene therapy using adeno-associated viruses (AAVs). Using AAVs as vectors to replace the non- or dysfunctional gene in vivo is a relatively simple model which has created an unprecedented opportunity for the future of NDD treatment. Advances...

... from any ideas, methods, instructions or products referred to in the content or advertisements. Lentiviral vector Cerebellum Purkinje cells Oligodendrocytes Granule neurons Gene therapy Original Paper Dev Neurosci 2006;28:216 221 DOI: 10.1159/000091919 Received: August 3, 2005 Accepted...

... advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements. Brain Glioma Gene therapy Virus vectors...