Abstract: A method for in utero genome editing, the method comprising administering to a subject an adenoviral vector, wherein the subject is a fetus, the adenoviral vector comprising CRISPR-mediated base editor and a guide RNA (gRNA), the gRNA targeting a mutation in a therapeutic gene; and introducing a modified codon in the therapeutic gene by base editing the therapeutic gene, wherein the base editing is performed by the adenoviral vector, an adeno-associated viral vector or lipid based nanoparticle.

Abstract: A method for in utero and postnatal genome editing of a lysosomal storage disease gene, the method comprising administering to a subject an ABE or CBE complex, wherein the subject is an embryo, a fetus, a neonate, a child or an adult, ABE or CBE complex comprising CRISPR-mediated base editor and a guide RNA (gRNA), the gRNA targeting a mutation in a therapeutic gene; and introducing a modified codon in the therapeutic gene by base editing the therapeutic gene without inducing double strand DNA breaks, wherein the base editing is performed by the adenoviral vector, an adeno-associated viral vector, nucleoprotein complex or an mRNA in a lipid based nanoparticle.

Abstract: Compositions and methods for in utero gene editing in mammalian lung cells are disclosed.