It is finally all coming together… My career in reproductive biology and AIDS began in 1983, with the goal of understanding the influence of viruses on early embryo development. Wonderful young scientists joined my laboratory for periods of training during the ensuing 33 years, and together we have made many discoveries that bring our laboratory skills to where they are today. We now have the foundation to begin to generate off-the-shelf stem cells for everybody.
Additionally, because we are a nimble institution, we were able to quickly change research direction to take advantage of a new technology, reported in 2013, that allows unprecedented precision in silencing genes. This advance has two immediately practical applications for our “off-the-shelf” stem cell research goals:
(1) It is now feasible to specifically silence the genes responsible for the proteins on cells that cause immune rejection. Just as Type “O” blood can be administered to almost everyone, such a neutralized cell could be transplanted into many individuals without leading to immune rejection. This would be a major step forward in generating “off-the-shelf” stem cells for everybody. Our successful experiments in mouse eggs pave the way to translate the work to stem cells from human eggs. Like blood banks, such a stem cell bank could be available in emergency rooms for acute treatments, such as heart attack, stroke and spinal cord injury.
(2) It is also feasible to replicate the natural mutation in 1% of humans that renders individuals resistant to infection by HIV, the virus that causes AIDS. The ability to precisely silence this gene without causing other changes in the cell, in the same way it is naturally inactivated in 1% of humans, paves the way to deriving a library of stem cells resistant to HIV infection. IF those cells can be developed into bone marrow stem cells, and IF those bone marrow stem cells will function normally, they could be utilized as a powerful treatment, perhaps a cure, for HIV disease.
To help guide the work, we have meritorious individuals serving as our Ethics Advisory Board, our Human Subjects Committee and our Stem Cell Research Oversight Committee. Their guidance has allowed us to forge ahead into areas of stem cell development that larger institutions have shied away from because the work cannot be funded by our federal government. The “Dickey-Wicker Amendment” to the budget of the National Institutes of Health is renewed annually and prohibits funds to be used for studies of unfertilized human eggs. We have for years believed unfertilized eggs (“parthenotes”) will be a broadly applicable source of human stem cells.
Human egg research MUST be privately funded, progress depends entirely on private donations. No federal dollars can be used to study activated human eggs or parthenote stem cells. BRF is uniquely positioned to push this exciting field forward! Thank you for your continued support.
Ann A Kiessling, PhD
Director, Bedford Research Foundation