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Novarum Scholar — Research Digest
New papers in your keyword
Nature
High-fidelity prime editing in human hematopoietic stem cells
Researchers demonstrate a refined prime editing approach achieving over 90% efficiency in HSCs with minimal off-target effects.
bioRxivPREPRINT
Compact Cas9 variants enable efficient in vivo delivery
A new family of miniaturized Cas9 nucleases fits within AAV packaging limits while maintaining editing efficacy.
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Novarum Scholar — Research Digest
New papers in CRISPR gene editing
Tuesday, April 21, 2026  ·  4 new articles
Nature Biotechnology· Apr 19, 2026OPEN ACCESS
High-fidelity prime editing in human hematopoietic stem cells
Chen Y., Liu R., Park S. et al.
We present a refined prime editing approach that achieves over 90% efficiency in HSCs while maintaining genomic stability across 2,400 off-target sites analyzed.
AI Summary
A major step toward CRISPR-based therapies for blood disorders. The team's optimized prime editing system works in the exact cell type affected by sickle cell disease and beta-thalassemia — with minimal off-target cuts.
Read paper →
bioRxiv· Apr 20, 2026PREPRINT
Compact Cas9 variants enable efficient in vivo delivery via AAV vectors
Hoffmann M., Saito K., Diaz A. et al.
We engineer three Cas9 orthologs under 3.2 kb that maintain high specificity in liver, muscle, and retinal tissue across four mouse models.
AI Summary
AAV packaging limits have been a hard wall for in vivo CRISPR therapy. This preprint reports compact Cas9s that fit inside standard AAV capsids while matching full-size Cas9 editing rates — potentially unlocking systemic delivery.
Read paper →
Cell· Apr 17, 2026RESTRICTED
Base editing corrects F508del CFTR mutation in intestinal organoids with 99.7% precision
Torres-Ayala L., Singh P., Watanabe N. et al.
Using adenine base editing, we correct the most prevalent cystic fibrosis mutation across patient-derived intestinal organoids with near-complete precision and no detectable indels.
AI Summary
Cystic fibrosis affects ~70% of patients via one mutation. This study shows base editing — a gentler form of CRISPR — can fix it in organoids without cutting the DNA strand, which means fewer unintended edits.
Read paper →
+ 1 more article in this issue
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