Table.
Summary of patient demographics and nystagmus
| Low dose | Medium dose | High dose | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| NP01 | NP02 | NP03 | NP04 | CH06* | CH08 | CH09 | CH10 | CH11 | CH12 | CH13 | NP15 | |
| Date at administration | Oct 11, 2007 |
Dec 13, 2007 |
Jan 24, 2008 |
April 10, 2008 |
May 22, 2008 |
July 10, 2008 | Sept 25, 2008 | Nov 18, 2008 | Dec 30, 2008 |
March 5, 2009 |
April 16, 2009 |
June 2, 2009 |
| Age (years) | 26 | 26 | 19 | 17 | 20 | 9 | 8 | 10 | 24 | 44 | 35 | 11 |
| Sex | Female | Male | Female | Male | Female | Male | Male | Male | Female | Female | Male | Male |
| RPE65 mutations | E102K/ E102K |
E102K/ E102K |
R234X/ R234X |
R91W/ T149N |
IVS1+5g>a/ L341S |
F530fs/F530fs | R124X/ Lys297del1aggA |
IVS1+5g>a/ Phe530del1ttc |
V473D/ V473D |
K303X/ W431C |
IVS1+5g>a/ IVS1+5g>a |
D167W/ H313R |
| Injected eye† | Right | Right | Right | Left | Right | Right | Left | Right | Right | Right | Right | Right |
| Nystagmus frequency before and 90 days after gene therapy (Hz) |
3·8/2·0 | 2·3/2·1 | 4·2/3·0 | 1·0/0 | 1·1/1·3 | 3·7/2·0 | 2·5/2·0 | 4·0/2·0 | 0·5/0·5 | 1·0/0·25 | 0·3/0·4 | 0·4/0·4 |
*
Also heterozygous for RDH12 S203R.
†
Eye with worst sight selected for surgery.