Gene therapy
Recent articles
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.
Latest ‘prime-editing’ tools tackle delivery, safety issues
The gene-editing advances make it easier to target specific tissues in mice and detect off-target effects.
Latest ‘prime-editing’ tools tackle delivery, safety issues
The gene-editing advances make it easier to target specific tissues in mice and detect off-target effects.
RNA therapy restores gene function in monkeys modeling Angelman syndrome
The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.
RNA therapy restores gene function in monkeys modeling Angelman syndrome
The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.
Explore more from The Transmitter
Are Brains and AI Converging?—an excerpt from ‘ChatGPT and the Future of AI: The Deep Language Revolution’
In his new book, to be published next week, computational neuroscience pioneer Sejnowski tackles debates about AI’s capacity to mirror cognitive processes.
Are Brains and AI Converging?—an excerpt from ‘ChatGPT and the Future of AI: The Deep Language Revolution’
In his new book, to be published next week, computational neuroscience pioneer Sejnowski tackles debates about AI’s capacity to mirror cognitive processes.
New tissue-clearing techniques let microscopes peer deeper into living brains
Washing mouse brain tissue with a blood protein or complex sugar can illuminate cells 550 micrometers into the cortex without compromising its normal physiology.
New tissue-clearing techniques let microscopes peer deeper into living brains
Washing mouse brain tissue with a blood protein or complex sugar can illuminate cells 550 micrometers into the cortex without compromising its normal physiology.
New catalog charts familial ties from autism to 90 other conditions
The research tool reveals associations stretching across three generations.
New catalog charts familial ties from autism to 90 other conditions
The research tool reveals associations stretching across three generations.