Appeal No. 2006-1879 Page 2
Application No. 10/010,114
within the cell by the reticuloendothelial system; and transport to and through the
nuclear membrane into the nucleus of the cell where transcription of the genetic
material can take place.” Specification, page 2, lines 5-14. The specification discloses
a “multifunctional molecular complex for the transfer of a nucleic acid composition to a
target cell comprising . . . : 1) said nucleic acid composition; 2) one or more cationic
polyamine components . . . ; [and] 3) one or more endosome membrane disruption
promoting components.” Page 12, lines 2-9.
“The core, or backbone[,] of the transfer moiety is the cationic polyamine,
containing between 3 and 12 amines.” Page 23, lines 17-18. The function of the
cationic polyamine is “to overcome the incompatibility arising from the hydrophilic nature
of the nucleic acid molecule and the lipophilic nature of the cell membrane.” Id., lines
20-23.
“The next component of the transfer moiety is the endosome membrane
disruption promoting component. . . . This can either comprise one or more lipophilic
long chain alkyl groups attached through one or more of the nitrogen atoms of said
polyamine, or can comprise a bridging group . . . through which there is attached a
fusogenic peptide, or cholic acid or cholesteryl or derivative compound.” Page 25,
lines 28-37. This component “prevent[s] degradation of the nucleic acid molecule in a
lysosome,” page 16, lines 27-28, by “permit[ting] the complex to escape from the
endosome, whereupon it can migrate into the nucleus of the target cell, and release the
nucleic acid composition, whose genetic information can then be transcribed within said
nucleus.” Page 34, lines 2-6.
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