Fatwa Adikusuma

This paper develops a genetically engineered trophoblast organoid model of preeclampsia that recapitulates molecular and functional features of the disease.

This study shows that shortening the 4T poly-T tract in gRNA scaffolds improves transcript levels and editing efficiency, especially under low vector conditions, for both SpCas9 and SaCas9.

This study uses gene-edited placental organoids to show that ACE2 knockout causes delayed growth and increased cell death, while the ACE2 rs2074192 TT genotype increases ACE2 expression but reduces enzyme activity.

The paper provides a protocol for generating and characterizing mouse models of Duchenne Muscular Dystrophy using CRISPR-Cas9 and describes methods for phenotypic and molecular analysis.

The paper generates a single-copy humanized DMD mouse model by deleting a duplicated transgene to improve the accuracy of CRISPR therapy testing.

This study demonstrates that antisense oligonucleotide therapy reduces seizures and improves survival in a KCNT1 mouse model of encephalopathy.

The paper investigates the role of the Nestin neural enhancer in regulating Nes expression during embryonic development and shows it is not essential for embryo viability.

This paper develops PEA1 plasmids and PE-nuclease systems for efficient prime editing in human and mouse cells, achieving high editing efficiency in some cell lines and fetal mice.