Sharan Janjuha

RNA-LNP–mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I

András Tálas, Eleonora I. Ioannidi, Yanik Weber, Tatjana Haenggi et autres

Citrullinemia type I (CTLN1) is a severe urea cycle disorder caused by pathogenic variants in the ASS1 (argininosuccinate synthetase 1) gene, for which liver transplantation remains the only curative option. Here, we used prime editing to correct the Ass1 fold mouse model …

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RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I

András Tálas, Eleonora I. Ioannidi, Yanik Weber, Tatjana Haenggi et autres

Citrullinemia type I (CTLN1) is a severe urea cycle disorder caused by pathogenic variants in the (argininosuccinate synthetase 1) gene, for which liver transplantation remains the only curative option. Here, we used prime editing to correct the mouse model of CTLN1. Adeno-associated …

Prime editing of the β1 adrenoceptor in the brain restores physiological REM sleep in a mouse model of Alzheimer’s disease

Desirée Böck, Lisa Tidecks, Maria Wilhelm, Waleed ElGrawani et autres

Abstract Prime editing offers versatile genome modifications with therapeutic potential; yet its use to modulate neural circuitry remains underexplored. Here, we used adeno-associated viral vectors to deliver prime editors into the mouse brain and introduced the naturally occurring Adrb1 A187V variant of …

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Prime editing of the β1 adrenoceptor in the brain restores physiological REM sleep in a mouse model of Alzheimer’s disease

Desirée Böck, Lisa Tidecks, Maria Wilhelm, Waleed ElGrawani et autres

Prime editing offers versatile genome modifications with therapeutic potential; yet its use to modulate neural circuitry remains underexplored. Here, we used adeno-associated viral vectors to deliver prime editors into the mouse brain and introduced the naturally occurring Adrb1 $^{A187V}$ variant of the …

Prime editing of the β1 adrenoceptor in the brain restores physiological REM sleep in a mouse model of Alzheimer’s disease

Desirée Böck, Lisa Tidecks, Maria Wilhelm, Waleed ElGrawani et autres

Prime editing offers versatile genome modifications with therapeutic potential; yet its use to modulate neural circuitry remains underexplored. Here, we used adeno-associated viral vectors to deliver prime editors into the mouse brain and introduced the naturally occurring Adrb1 $^{A187V}$ variant of the …

Spatial profiling of gene editing by in situ sequencing in mice and macaques

Sharan Janjuha, Tatjana Haenggi, Thomas C. Chamberlain, Tanja Rothgangl et autres

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Spatial profiling of gene editing by in situ sequencing in mice and macaques

Sharan Janjuha, Tatjana Haenggi, Thomas C. Chamberlain, Tanja Rothgangl et autres

Predicting adenine base editing efficiencies in different cellular contexts by deep learning

Lucas Kissling, Amina Mollaysa, Sharan Janjuha, Nicolas Mathis et autres

BACKGROUND: Adenine base editors (ABEs) enable the conversion of A•T to G•C base pairs. Since the sequence of the target locus influences base editing efficiency, efforts have been made to develop computational models that can predict base editing outcomes based on the …

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Predicting adenine base editing efficiencies in different cellular contexts by deep learning

Lucas Kissling, Amina Mollaysa, Sharan Janjuha, Nicolas Mathis et autres

Background: Adenine base editors (ABEs) enable the conversion of A•T to G•C base pairs. Since the sequence of the target locus influences base editing efficiency, efforts have been made to develop computational models that can predict base editing outcomes based on the …

Publisher Correction: Machine learning prediction of prime editing efficiency across diverse chromatin contexts

Nicolas Mathis, Ahmed Allam, András Tálas, Lucas Kissling et autres

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Machine learning prediction of prime editing efficiency across diverse chromatin contexts

Nicolas Mathis, Ahmed Allam, András Tálas, Lucas Kissling et autres

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Predicting prime editing efficiency across diverse edit types and chromatin contexts with machine learning

Nicolas Mathis, Ahmed Allam, András Tálas, Elena Benvenuto et autres

Abstract Prime editing is a powerful genome editing technology, but its efficiency varies depending on the pegRNA design and target locus. Existing computational models for predicting prime editing rates are limited by their focus on specific edit types and by omitting the …

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