New Funding Brings Personalized CRISPR Cures Closer to Children with Rare Diseases

Multiplex, single-cell CRISPRa screening for cell type specific regulatory elements

CRISPR-based gene activation (CRISPRa) is a strategy for upregulating gene expression by targeting promoters or enhancers in a tissue/cell-type specific manner. Here, we describe an experimental framework that combines highly multiplexed perturbations with single-cell RNA sequencing (sc-RNA-seq) to identify cell-type-specific, CRISPRa-responsive cis-regulatory elements and the gene(s) they regulate. Random combinations of many gRNAs are introduc…

New funding brings personalized CRISPR cures closer to children with rare diseases

Personalized CRISPR cures for children born with rare genetic diseases are now a step closer to being more widely available.

Chan Zuckerberg Initiative, Innovative Genomics Institute Announce New Center for Pediatric CRISPR Cures

SAN FRANCISCO, July 8, 2025 /PRNewswire/ -- Personalized CRISPR cures for children born with rare genetic diseases are now a step closer to being more widely available. Today, the Chan Zuckerberg Initiative (CZI) and the Innovative Genomics Institute (IGI) announced…

Center for Pediatric CRISPR Therapies Funded by IGI, CZI

The recent clinical success of treating “Baby KJ” Muldoon—an infant born with a rare metabolic disease—with the first-ever personalized gene-editing therapy brought much-needed enthusiasm to the genome editing world. It also raised a lot of questions: how will this advance develop beyond an n-of-1 therapy? How will genome editing strategies be developed more broadly and brought to more children? Now, the Chan Zuckerberg Initiative (CZI) and the …

Law Targets Rare Pediatric Diseases

A key lawmaker and Florida State University leaders Wednesday touted a new law that includes offering free genetic screening for newborns and bolstering research on rare pediatric diseases.

Ultrasensitive CRISPR-based biosensing platform for early diagnosis of ...