Gene therapy
Recent articles
Gene replacement therapy normalizes some traits in SYNGAP1 model mice
The first published virus-based gene therapy for SYNGAP1 deletion yields benefits despite the gene’s long length and complexity.
Gene replacement therapy normalizes some traits in SYNGAP1 model mice
The first published virus-based gene therapy for SYNGAP1 deletion yields benefits despite the gene’s long length and complexity.
Boosting SCN2A expression reduces seizures in mice
A modified form of CRISPR amps up expression of the gene—a strategy that could apply to other gene variations linked to autism.
Boosting SCN2A expression reduces seizures in mice
A modified form of CRISPR amps up expression of the gene—a strategy that could apply to other gene variations linked to autism.
Expediting clinical trials for profound autism: Q&A with Matthew State
Aligning Research to Impact Autism, a new initiative funded by the Sergey Brin Family Foundation, wants to bring basic science discoveries to the clinic faster.
Expediting clinical trials for profound autism: Q&A with Matthew State
Aligning Research to Impact Autism, a new initiative funded by the Sergey Brin Family Foundation, wants to bring basic science discoveries to the clinic faster.
Split gene therapy delivers promise in mice modeling Dravet syndrome
The new approach overcomes viral packaging limitations by delivering SCN1A piecemeal and stitching it together in target cells.
Split gene therapy delivers promise in mice modeling Dravet syndrome
The new approach overcomes viral packaging limitations by delivering SCN1A piecemeal and stitching it together in target cells.
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.
Explore more from The Transmitter
Portfolio of SCN2A gene variants, and more
Here is a roundup of autism-related news and research spotted around the web for the week of 9 March.
Portfolio of SCN2A gene variants, and more
Here is a roundup of autism-related news and research spotted around the web for the week of 9 March.
Hippocampus builds reputation as ‘general-purpose statistical learning machine’
New cross-species findings may help settle a long-standing debate about whether the hippocampus is required for passive learning.
Hippocampus builds reputation as ‘general-purpose statistical learning machine’
New cross-species findings may help settle a long-standing debate about whether the hippocampus is required for passive learning.
‘The Fox, the Shrew, and You: How Brains Evolved,’ an excerpt
In his new book, Rogier Mars provides a detailed account of animal and human brain evolution. In this excerpt from Chapter 1, he starts with the sea squirt—and why it needs the brain it eats after its larval stage.
‘The Fox, the Shrew, and You: How Brains Evolved,’ an excerpt
In his new book, Rogier Mars provides a detailed account of animal and human brain evolution. In this excerpt from Chapter 1, he starts with the sea squirt—and why it needs the brain it eats after its larval stage.