Development
of Human Gene Therapy Protocols
at the
University
of Florida
Phase
I trials are primarily
done to determine safety,
that is, insure that the
dose used in clinical applications
is not toxic or have side
effects that would preclude
the therapy's use in human
patients. If correctly
designed, a Phase I trial
can also yield information
about how effective the
therapy will be.
Cystic
fibrosis
Phase
I and Phase II
trials of aerosolized
AAV-CFTR are
currently ongoing.
For further information,
contact Margaret Humphries.
Alpha
1 Alpha-1-antitrypsin
Deficiency
A
phase 1 trial
will begin early
in 2004. For
further information,
contact Margaret
Humphries. |