Pablo Pérez‐Piñera

In vivo CRISPR base editing for treatment of Huntington's disease

This study uses CRISPR base editing to create HTT protein isoforms resistant to caspase-6 cleavage, reducing toxicity and neurodegeneration in a rodent HD model.

Intron Retention Controls Localization of lncRNAs PURPL and MALAT1 to Promote Cell Proliferation and Migration

The paper identifies U2AF2's role in intron retention of lncRNAs PURPL and MALAT1, showing how this process affects their localization and promotes cell proliferation and migration.

Chromatin-associated lncRNA-splicing factor condensates regulate hypoxia responsive RNA processing of genes pre-positioned near nuclear speckles

The paper shows that MALAT1 long noncoding RNA forms condensates with splicing factors to regulate hypoxia-induced alternative splicing near nuclear speckles.

SPLICER: A Highly Efficient Base Editing Toolbox That Enables In Vivo Therapeutic Exon Skipping

SPLICER improves exon skipping by editing both splice acceptor and donor sites, reducing aberrant splicing and enabling therapeutic applications in Alzheimer's disease.

Chromatin structure and context-dependent sequence features control prime editing efficiency

This study identifies epigenomic and sequence features that influence prime editing efficiency and provides guidelines for optimizing prime editor designs.

Epigenetic engineering of yeast reveals dynamic molecular adaptation to methylation stress and genetic modulators of specific DNMT3 family members

This study uses yeast to investigate how DNA methylation is regulated by DNMT3 family members and identifies sequence preferences and spatial patterns of methylation.