Gene therapy
Recent articles
Molecular changes after MECP2 loss may drive Rett syndrome traits
Knocking out the gene in adult mice triggered up- and down-regulated expression of myriad genes weeks before there were changes in neuronal function.
Molecular changes after MECP2 loss may drive Rett syndrome traits
Knocking out the gene in adult mice triggered up- and down-regulated expression of myriad genes weeks before there were changes in neuronal function.
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.
Explore more from The Transmitter
Newly awarded NIH grants for neuroscience lag 77 percent behind previous nine-year average
Since President Donald Trump took office on 20 January, the National Institute of Neurological Disease and Stroke and the National Institute of Mental Health have awarded one quarter as many new grants as during the same two-month period, on average, since 2016.
Newly awarded NIH grants for neuroscience lag 77 percent behind previous nine-year average
Since President Donald Trump took office on 20 January, the National Institute of Neurological Disease and Stroke and the National Institute of Mental Health have awarded one quarter as many new grants as during the same two-month period, on average, since 2016.
Releasing the Hydra with Rafael Yuste
Losing HHMI Investigator status caused Yuste to study neural networks in a new way.
Releasing the Hydra with Rafael Yuste
Losing HHMI Investigator status caused Yuste to study neural networks in a new way.
Coding error caused layoffs at National Institute of Neurological Disorders and Stroke this week, source says
Thirty employees—including 11 lab heads—at the institute should “immediately return to work,” according to an email the institute’s Office of Human Resources sent to top administration at the institute Wednesday evening.
Coding error caused layoffs at National Institute of Neurological Disorders and Stroke this week, source says
Thirty employees—including 11 lab heads—at the institute should “immediately return to work,” according to an email the institute’s Office of Human Resources sent to top administration at the institute Wednesday evening.