Attachment VI - Page 1 
3 
POINTS CF CONSIDER IN THE DESIGN AND SUBMISSION CF HUMAN 
SCMATIC-CELL OHE THERAPY PROTOCOLS 
Introduction 
Experiments in which recombinant ENA 1 is introduced into cell of a human 
subject with the intent of stably modifying the subject's genome are covered by 
Section III-A— 4 of the National Institutes of Health (NTH) Guidelines for 
Research Involving Recombinant ENA Molecules (49 FR 46266). Section III-A-4 
requires such experiments to be reviewed by the NIH Recombinant DIA Advisory 
Ccmmittee (RAC) and approved by the NIH. RAC consideration of each proposal 
will be on a case-by-case basis and will follow publication of a precis of the 
proposed, m the Federal Register , an opportunity for public comment, and review 
of the proposed, by the working group of the RAC. RAC recommendations on each 
proposed will be forwarded to the NIH [Director for a decision, vhich will then 
by published in the Federal Register . In accordance with Section IV-C-l-b of 
the NIH Guidelines, the NIH Director may appro/e proposals only if he finds 
that they present "no significant risk to health or the environment." 
In general, it is expected that somatic-cell gene therapy protocols will not 
present a risk bo the envirorment as the recombinant DNA is expected bo be 
confined bo the human subject. Nevertheless, item I-B-4-b of the "Points to 
Consider" document asks the researchers to eddress specifically this point. 
^-Experiments using retroviruses (RNA) as vectors cure also covered by the NIH 
Guidelines for Research Involvinq Recombinant DNA Molecules and hence by this 
document. Section III-A— 4 applies to both recombinant DNA and DNA derived 
from recombinant FNA. 
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