Patent ID: 7217571

Claim:
A method for replacing a target fragment of a gene in a cell in vitro, the method comprising delivering to the cell an exogenous replacement DNA fragment, the replacement DNA fragment consisting of: (a) at least one replacement exon having a 3′ end and a 5′ end; (b) a 3′ end consisting of a 3′ flanking noncoding sequence adjacent to the 3′ end of the at least one replacement exon; and (c) a 5′ end consisting of a 5′ flanking noncoding sequence adjacent to the 5′ end of the at least one replacement exon; wherein the replacement DNA fragment is from 1 to about 2000 bases and includes less than all of the exons of the gene, and wherein the 3′ flanking noncoding sequence of the replacement DNA fragment is homologous to and anneals to a 3′ flanking noncoding sequence adjacent to the target fragment, and the 5′ flanking noncoding sequence of the replacement DNA fragment is homologous to and anneals to a 5′ flanking noncoding sequence adjacent to the target fragment, so that the exogenous replacement DNA fragment replaces the target fragment of the gene in the cell, wherein the DNA is delivered into the cell by microinjection or electroporation, or by local direct administration of a lipid-DNA complex comprising the replacement DNA fragment.