Author

Fawad Ur Rehman

2 papers indexed here

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Open access Sep 2026

Prime Editing Corrects the HBB Codon 8/9 (+G) Mutation in Patient-Derived Induced Pluripotent Stem Cells and Restores β-Globin Expression in iPSC-Derived Erythroid Cells

Background Homozygosity for the HBB codon 8/9 (+G) frameshift (c.27dup (p.Ser10ValfsTer14)) causes transfusion-dependent β⁰-thalassaemia and is common in South Asia. Prime editing can reverse this insertion without double-strand breaks or donor DNA, but its efficiency depends on pegRNA design. Methods We derived Sendai...

Irfan Hussain, Kainaat Mumtaz, Maliha Javed et al. · bioRxiv · 0 citations

Open access Jul 2026

A TLS11a-decorated ionizable lipid nanoparticle platform and a multilevel-validated CRISPR LDLR-knockout HepG2 model for hepatocyte-preferential mRNA delivery

A modular aptamer-guided LNP platform to enable hepatocyte-selective genome editing through an LDLR-independent route is designed and validated and a validated LDLR-null hepatocyte model is validated for studying LDLR-dependent biology and disease.

Irfan Hussain, Naji Kholaif, Raghad Alsultan et al. · bioRxiv · 0 citations