Page 91 - Ethical Guidelines for Conducting Research Studies Involving Human Subjects

theory, be highly effective in counteracting genetic disorders and hereditary
diseases, some jurisdictions, including Australia, Canada, Germany, Israel,
Switzerland, and the Netherlands prohibit this for application in human beings,
at least for the present, for technical and ethical reasons, including insufficient
knowledge about possible risks to future generations and higher risk than somatic
gene therapy (e.g. using non-integrative vectors). The USA has no federal
legislation specifically addressing human germ-line or somatic genetic
modification (beyond the usual FDA testing regulations for therapies in general).

2) Somatic gene therapy

(NHMRC, Australia) Somatic cell gene therapy involves the introduction of DNA
(deoxyribonucleic acid) or RNA (ribonucleic acid) into the somatic (non-
reproductive) cells of humans, or the introduction into humans of cells whose
genetic material has been modified, in order to provide an alternative form of
treatment to improve the health of individuals. It involves the use of a gene
carrier or ‘vector’ (often a defective virus) to carry a gene into cells with a view to
integration of the gene into chromosomal DNA and its long term expression.
However, additional novel and varied strategies for introducing or modifying gene
expression in humans have been devised which make the boundary between gene
therapy and other treatment strategies hazy. Consider immunization with a virus
expressing a particular protein, or immunization with naked DNA, to treat or
prevent a chronic viral infection such as HIV, or as part of cancer treatment.
These methodologies overlap with the traditional concept of gene therapy in that
DNA is introduced into somatic cells, but could also be thought of as modifications
to standard immunization strategies in which DNA, rather than protein, is used
to generate the immune response. Sometimes there may be uncertainty about
whether a proposed research project should be considered to fall under the
heading of ‘gene therapy’. Where there is doubt as to whether a research proposal
falls into the category of gene therapy, researchers should seek guidance from the
BMRC.

DNA or RNA can also be introduced into somatic cells to mark their distribution
and fate. Although no therapeutic benefit is expected, such studies are important
for understanding the underlying basis for diseases or as first steps in the
development of some somatic cell gene therapies for serious diseases. There may
be other justifiable therapeutic or non-therapeutic reasons for introducing DNA or
RNA into human somatic cells. While the introduction of DNA or RNA into
somatic cells is ethically acceptable, the introduction of DNA or RNA into germ
(reproductive) cells or embryos is ethically unacceptable, since there is insufficient
knowledge about the possible consequences including hazards and effects on
future generations.

BMRC ETHICAL GUIDELINE ON HUMAN SUBJECTS Page 87