In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse modelRNA-SEQ DATADISEASE PROGRESSIONGENE DELIVERYHUMAN-CELLSALSDEGENERATIONMICEQUANTIFICATIONEFFICIENTDESIGNCRISPR-based gene editing technology represents a promising approach to ...
Ferroptosis represents a form of programmed cell death that is propelled by iron-dependent lipid peroxidation, thereby being distinguished by the prominent features of iron accumulation and lipid peroxidation. Ferroptosis has been implicated in numerous
Through combinatorial library design, novel ICLs have been identified like A6, 113-O12B, 16-I, 4A3-Cits, and BAmP-TK-12 for targeted delivery to specific cells and improved mRNA delivery efficiency (Fig. 3A) [51, 52]. For example, Anderson et al. developed A6, an ionizable lipid with...
Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 10, 2105–2111 (2003). CAS PubMed Google Scholar Duan, D. et al. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics ...
In vivo genome editing represents an emerging field in the treatment of monogenic disorders, as it may constitute a solution to the current hurdles in classic gene addition therapy, which are the low levels and limited duration of transgene expression. Following the introduction of a double strand...
ZFNs function as dimers by coupling DNA binding motifs from transcription factors with the FokI endonuclease domain to generate double-stand breaks at their target site. A typical genome editing approach is to target the disease locus itself; however, the proportion of alleles successfully edited may...
Retrotransposons are highly prevalent in most animals and account for more than 35% of the human genome. However, the prevalence, biogenesis mechanism and function of retrotransposons remain largely unknown. Here, we developed retroSeeker, a novel computational software that identifies novel retrotransposo...
Genome editing is transforming bioscience research, but its application to non-model organisms, such as farmed animal species, requires optimisation. Salmonids are the most important aquaculture species by value, and improving genetic resistance to infec
CRISPR/cas9 system is emerging as a novel genome editing tool in biology/medicine research, but its indication in microRNA research has not been studied exclusively. In this study, we clone CRISPR/cas9 constructs with single- guide RNAs specifically targeting biogenesis processing sites of selected ...
While we have focused here on the correction of an exon 50 deletion on the restoration of dystrophin expression by AAV9-mediated delivery of gene-editing components in vivo, the same approach can be taken for monitoring any other DMD mutations. The WT-Dmd-Luc mice will also be useful for ...