Gene therapy
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
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
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
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
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
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
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
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
Going on Trial: Orphan drugs; CBD; bumetanide
This month’s newsletter takes a close look at the orphan drug program in the United States, several cannabis-based therapies and a secondary analysis of bumetanide, among other new developments in autism-related drug trials.
Epigenome edits unmute MECP2 in Rett-like neurons
The approach removes methyl tags from the gene and shields it from other silencing factors without changing the gene itself, raising hopes for a new treatment.
Epigenome edits unmute MECP2 in Rett-like neurons
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Robots boost data consistency in rodent studies reliant on mechanical, optogenetic stimulation
Two new devices take experimenter variation out of the equation, the lead investigators say.
Robots boost data consistency in rodent studies reliant on mechanical, optogenetic stimulation
Two new devices take experimenter variation out of the equation, the lead investigators say.
Spina bifida; MDMA effects in a mouse model of autism; maternal autoantibodies
Here is a roundup of autism-related news and research spotted around the web for the week of 13 May.
Spina bifida; MDMA effects in a mouse model of autism; maternal autoantibodies
Here is a roundup of autism-related news and research spotted around the web for the week of 13 May.
Autism research is becoming more diverse but not yet more global
Hosting the International Society for Autism Research annual meeting—which starts tomorrow—in Australia takes a step in that direction, making it easier for researchers, clinicians and advocates from the Western Pacific Region to participate.
Autism research is becoming more diverse but not yet more global
Hosting the International Society for Autism Research annual meeting—which starts tomorrow—in Australia takes a step in that direction, making it easier for researchers, clinicians and advocates from the Western Pacific Region to participate.