Results 111 to 120 of about 515,050 (293)

Double-strand break-free and transgene-free genome editing in the microalga Nannochloropsis oceanica using removable vectors containing the CRISPR base editing system

Scientific Reports
The accumulation of lipids by algae makes them attractive for carbon-neutral fuel production; however, the industrial-scale production of algal lipids has yet to be achieved.
Keishi Moroi, Takashi Yamamoto, Tomokazu Kurita   +2 more
doaj   +1 more source

PARK(ing) time–How park deficiency affects the biological clock in a Drosophila model of Parkinson's disease

FEBS Letters, EarlyView.
Drosophila park mutants serve as a model for Parkinson's disease. We used this strain to investigate the connection between oxidative stress and the circadian clock mechanism. We showed that increased oxidative stress affects the physiology of pacemaker cells, disrupting their daily structural plasticity. Lack of rhythmic signaling from pacemaker cells
Kamila Zientara, Kinga Skoczek, Elzbieta Pyza, Milena Damulewicz   +3 more
wiley   +1 more source

Gene Editing in Fabry Disease: A Strategy Delineation

, 2022
Oral presentation by first authorThe use of iPSCs, in the last years became wide spread, even in our group at INSA, the use of iPSCs to develop models of disease is now envisaged for various Lysosomal Storage Diseases.
Moreira, Luciana, Amaral, Olga, Duarte, Ana J., Ribeiro, Diogo   +3 more
core  

Breaking genetic shackles: The advance of base editing in genetic disorder treatment

Frontiers in Pharmacology
The rapid evolution of gene editing technology has markedly improved the outlook for treating genetic diseases. Base editing, recognized as an exceptionally precise genetic modification tool, is emerging as a focus in the realm of genetic disease therapy.
Fang Xu, Fang Xu, Caiyan Zheng, Caiyan Zheng, Weihui Xu, Weihui Xu, Shiyao Zhang, Shiyao Zhang, Shanshan Liu, Shanshan Liu, Xiaopeng Chen, Xiaopeng Chen, Kai Yao, Kai Yao   +13 more
doaj   +1 more source

α‐Synuclein aggregation landscape from phase separation to neurotoxic intermediates

FEBS Letters, EarlyView.
Alpha‐synuclein aggregation in Parkinson's disease involves a complex landscape of transient intermediates, including oligomers, fibrils and liquid–liquid phase separation (LLPS). A view is emerging in which LLPS maturation into solid‐like condensates may contribute to the formation of neurotoxic species.
Silvia Arino, Giuliana Fusco, Alfonso De Simone   +2 more
wiley   +1 more source

Recent advances in DNA-free editing and precise base editing in plants

, 2017
Genome-editing technologies based on the CRISPR (clustered regularly interspaced short palindromic repeat) system have been widely used in plants to investigate gene function and improve crop traits.
Yi Zhang, Caixia Gao
core   +1 more source

Deep learning models incorporating endogenous factors beyond DNA sequences improve the prediction accuracy of base editing outcomes

Cell Discovery
Adenine base editors (ABEs) and cytosine base editors (CBEs) enable the single nucleotide editing of targeted DNA sites avoiding generation of double strand breaks, however, the genomic features that influence the outcomes of base editing in vivo still ...
Tanglong Yuan, Leilei Wu, Shiyan Li, Jitan Zheng, Nana Li, Xiao Xiao, Haihang Zhang, Tianyi Fei, Long Xie, Zhenrui Zuo, Di Li, Pinzheng Huang, Hu Feng, Yaqi Cao, Nana Yan, Xinming Wei, Lei Shi, Yongsen Sun, Wu Wei, Yidi Sun, Erwei Zuo   +20 more
doaj   +1 more source

Modelling stem cell differentiation related processes—A practical overview for biologists

FEBS Letters, EarlyView.
Stem cell differentiation is complex and difficult to control experimentally. This review introduces suitable computational modelling approaches that can support stem cell research, from mechanistic ODE and abstract models to multiscale and deep learning methods.
Ricco Zeegelaar, Xinqi Yan, Lucas Jansen Klomp, Stefano Schivo, Janine N. Post   +4 more
wiley   +1 more source

In Vivo Base Editing of Scn5a Rescues Type 3 Long QT Syndrome in Mice

reservedLa sindrome del QT lungo è una patologia genetica causata dalla mutazione di alcuni geni codificanti canali ionici del cuore, tra cui Scn5a. Questa sindrome causa aritmie cardiache e, nei casi più gravi, torsioni di punta e morte prematura dell’
GELAIN, MATTEO
core  

In vivo precision base editing to rescue mouse models of disease

Molecular Therapy: Nucleic Acids
CRISPR base editing enables precise, irreversible base conversions without inducing double-stranded breaks (DSBs) and has gained significant attention in recent years. By converting cytosine to thymine (C→T) or adenine to guanine (A→G), base editors (BEs)
Aaron Schindeler, Julian Chu, Christal Au-Yeung, Hsien-Yin Kao, Samantha L. Ginn, Alexandra K. O’Donohue   +5 more
doaj   +1 more source