Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …
genome editing technology opened the door to provide a versatile approach for treating …
Epigenome editing technologies for discovery and medicine
Epigenome editing has rapidly evolved in recent years, with diverse applications that
include elucidating gene regulation mechanisms, annotating coding and noncoding …
include elucidating gene regulation mechanisms, annotating coding and noncoding …
Developmental and epileptic encephalopathies: from genetic heterogeneity to phenotypic continuum
Developmental and epileptic encephalopathies (DEEs) are a heterogeneous group of
disorders characterized by early-onset, often severe epileptic seizures and EEG …
disorders characterized by early-onset, often severe epileptic seizures and EEG …
On-demand cell-autonomous gene therapy for brain circuit disorders
Several neurodevelopmental and neuropsychiatric disorders are characterized by
intermittent episodes of pathological activity. Although genetic therapies offer the ability to …
intermittent episodes of pathological activity. Although genetic therapies offer the ability to …
Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome
Z Han, C Chen, A Christiansen, S Ji, Q Lin… - Science translational …, 2020 - science.org
Dravet syndrome (DS) is an intractable developmental and epileptic encephalopathy
caused largely by de novo variants in the SCN1A gene, resulting in haploinsufficiency of the …
caused largely by de novo variants in the SCN1A gene, resulting in haploinsufficiency of the …
CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …
Precision medicine for genetic epilepsy on the horizon: Recent advances, present challenges, and suggestions for continued progress
The genetic basis of many epilepsies is increasingly understood, giving rise to the possibility
of precision treatments tailored to specific genetic etiologies. Despite this, current medical …
of precision treatments tailored to specific genetic etiologies. Despite this, current medical …
Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model …
Dravet syndrome (DS) is a developmental and epileptic encephalopathy caused by
monoallelic loss-of-function variants in the SCN1A gene. SCN1A encodes for the alpha …
monoallelic loss-of-function variants in the SCN1A gene. SCN1A encodes for the alpha …
Sodium channelopathies in neurodevelopmental disorders
The voltage-gated sodium channel α-subunit genes comprise a highly conserved gene
family. Mutations of three of these genes, SCN1A, SCN2A and SCN8A, are responsible for a …
family. Mutations of three of these genes, SCN1A, SCN2A and SCN8A, are responsible for a …
[HTML][HTML] High-capacity adenoviral vectors: expanding the scope of gene therapy
The adaptation of adenoviruses as gene delivery tools has resulted in the development of
high-capacity adenoviral vectors (HC-AdVs), also known, helper-dependent or “gutless” …
high-capacity adenoviral vectors (HC-AdVs), also known, helper-dependent or “gutless” …