Rula Zain's profile

Rula Zain

Principal Investigator

Karolinska Institute

I'm working on

8 months ago

Nucleic acids therapeutics fo tandem-repeat expansion diseases

About

Integrates human genetics, cell and molecular biology, and structural approaches to elucidate mechanisms of complex disease, enable biomarker discovery, and translate findings into gene- and cell-therapy strategies from target identification to preclinical validation.

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Karolinska Institute

CRISPR-based prevention of tandem-repeat expansions in Huntington's disease

A novel CRISPR-mediated genome editing approach targets the Huntingtin gene to prevent somatic repeat instability in Huntington's disease. This method uses modified guide RNAs to block repeat expansions in patient-derive...

TechnologyIn developmentUniversity

Rula Zain

Principal Investigator

Karolinska Institute

Work experience

Karolinska Institute

Principal Investigator

Top industry applications

Oncology & cancer therapeutics

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Cell BiologyGene TherapyGeneticsMolecular BiologyMolecular MedicineNeurodegeneration

Last updated Feb 2026