Gates Package — page 817
of 1375 pages
← p.816 p.818 → · this page in the original PDF · package
Key points
- In vivo HSC transduction of -YAC mice with an ABE8e expressing HDAd5/35++ vector is safe and results
in efficient and stable -globin reactivation in ~70% of erythrocytes at levels of 30% of human -globin.
- Therapeutic editing is achieved in thalassemia and Sickle Cell Disease patient CD34+ cells with HDAdEF1.ABE8e, leading to phenotypic correction in in vitro differentiated erythroid cells.
This is our OCR of the page, with running headers and footers removed. The
Committee's PDF
is authoritative; quote from it. Machine-readable, including the uncleaned
text: /api/page/gates/817
Records on this page
| Record | Date | Type | Pages |
|---|---|---|---|
| gates:email:00740 | 2022-09-27 | 813–819 |