Gene therapy
Recent articles
What’s next for brain-directed gene therapy after death in Neurogene trial
The incident highlights that viral vectors can trigger deadly immune responses even when delivered directly to the nervous system.
What’s next for brain-directed gene therapy after death in Neurogene trial
The incident highlights that viral vectors can trigger deadly immune responses even when delivered directly to the nervous system.
Expanding set of viral tools targets almost any brain cell type
Harmless viruses that encase short noncoding DNA elements called enhancers enable cell-type-specific gene delivery across the central nervous system in rodents and primates.
Expanding set of viral tools targets almost any brain cell type
Harmless viruses that encase short noncoding DNA elements called enhancers enable cell-type-specific gene delivery across the central nervous system in rodents and primates.
Leveraging the power of community to strengthen clinical trials for rare genetic syndromes
Families can become not only participants but champions of these research efforts.
Leveraging the power of community to strengthen clinical trials for rare genetic syndromes
Families can become not only participants but champions of these research efforts.
CRISPR gives autism-linked genes a boost, rescues functioning
A modified form of the gene-editing tool increases the expression of CHD8 and SCN2A, showing potential for autism therapies.
CRISPR gives autism-linked genes a boost, rescues functioning
A modified form of the gene-editing tool increases the expression of CHD8 and SCN2A, showing potential for autism therapies.
New RNA editor boasts increased versatility, safety
The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.
New RNA editor boasts increased versatility, safety
The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.
Mutation in top autism-linked gene may alter eye reflex
The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.
Mutation in top autism-linked gene may alter eye reflex
The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.
New template streamlines sharing clinical trial results
This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.
New template streamlines sharing clinical trial results
This month’s Going on Trial newsletter explores a new tool for communicating with clinical trial participants and their families, among other drug development news.
Compact kin of CRISPR discovered across eukaryotes
The newfound DNA-cutting enzyme, called Fanzor, can be programmed to edit the human genome and could prove easier to deliver to cells than current CRISPR tools.
Compact kin of CRISPR discovered across eukaryotes
The newfound DNA-cutting enzyme, called Fanzor, can be programmed to edit the human genome and could prove easier to deliver to cells than current CRISPR tools.
Spotted around the web: Synthetic embryos; Angelman gene therapy
Here is a roundup of news and research for the week of 26 June.
Spotted around the web: Synthetic embryos; Angelman gene therapy
Here is a roundup of news and research for the week of 26 June.
Going on Trial: Gene therapy for Rett; return to arbaclofen
This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.
Going on Trial: Gene therapy for Rett; return to arbaclofen
This month’s newsletter looks at the early safety data from the first gene therapy trial for Rett syndrome, among other drug development news.
Explore more from The Transmitter
The non-model organism “renaissance” has arrived
Meet 10 neuroscientists bringing model diversity back with the funky animals they study.
The non-model organism “renaissance” has arrived
Meet 10 neuroscientists bringing model diversity back with the funky animals they study.
Assembloids illuminate circuit-level changes linked to autism, neurodevelopment
These complex combinations of organoids afford a closer look at how gene alterations affect certain brain networks.
Assembloids illuminate circuit-level changes linked to autism, neurodevelopment
These complex combinations of organoids afford a closer look at how gene alterations affect certain brain networks.
Rajesh Rao reflects on predictive brains, neural interfaces and the future of human intelligence
Twenty-five years ago, Rajesh Rao proposed a seminal theory of how brains could implement predictive coding for perception. His modern version zeroes in on actions.
Rajesh Rao reflects on predictive brains, neural interfaces and the future of human intelligence
Twenty-five years ago, Rajesh Rao proposed a seminal theory of how brains could implement predictive coding for perception. His modern version zeroes in on actions.