Appeal No. 2001-0407 Page 4
Application No. 08/460,215
25-28. The 53,577 base-pair DNA sequence of the (unmutated) genomic PKD1
gene is disclosed in Figure 1 (SEQ ID NO:1).
The specification discloses that the PKD1 gene can be used
therapeutically. See page 23:
The present invention encompasses the treatment of PKD. . . . All
or part of the normal PKD1 gene . . . can be delivered to kidney
cells or other affected cells using a variety of known methods,
including e.g. liposomes, viral vectors, recombinant viruses, and the
like. The gene can be incorporated into DNA vectors that
additionally comprise tissue-specific regulatory elements, allowing
PKD1 expression in a tissue-specific manner. This approach is
feasible if a particular mutant PKD1 allele, when present in a single
copy, merely causes the level of the PKD1 protein to diminish
below a threshold level necessary for normal function; in this case,
increasing the gene dosage by supplementing with additional
normal copies of the PKD1 gene should correct the functional
defect.
(Emphasis added.) The specification provides no disclosure of APKD-causing
mutations in the PKD1 gene, nor does it provide evidence establishing the
mechanism by which a PKD1 mutation causes APKD. The specification provides
no working examples of APKD treatment, although a prophetic example of a
method of diagnosing APKD is provided (pages 35-37).
Discussion
1. Claims 39 and 47
The examiner rejected claims 39 and 47 on the basis that these claims
recite the use of “intronless nucleic acids [sic] sequences” that are
“derived” from the DNA of SEQ ID NO:1. However[,] the
specification fails to provide any support for the use of such DNA
for therapeutic use and also fails to provide any guidance in regard
to how such nucleic acids would be derived. Therefore, the as filed
specification fails to provide descriptive support for the invention as
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