Transduction buffer

Abstract

The invention relates to transduction compounds, buffers and methods for introducing molecules into cells. The invention also relates to methods of treatment, pharmaceutical compositions and other uses of the transduction compounds and buffers. The invention also relates to modified cells obtainable by the transduction compounds, buffers and methods of the invention.

1 - 47 . (canceled) 
     
     
         48 . A transduction buffer for transducing a protein into a cell comprising
 a transduction compound and a salt selected from a sodium, lithium, potassium, caesium or rubidium salt,   wherein the transduction compound is selected from compounds 42, 31, 45, 43, 34, 41, 40, 39, 33, 44, 22, 20, 03, 30, 15, 38, 35, 11, 10, 28, 37, 01, 29, 02, 21, 36, 26, 16, 04, 19, 23, 24, 12, 14, 13, 05, 08 and 27 in Table 1,   wherein the osmolality of the transduction buffer is at least 750 mOsm/kg, and   wherein the pH of the transduction buffer is between about 6 and about 8.   
     
     
         49 . The transduction buffer of  claim 48 , wherein the salt is sodium chloride. 
     
     
         50 . The transduction buffer of  claim 48 , wherein the transduction compound is NDSB-201 (compound 01 in Table 1). 
     
     
         51 . The transduction buffer of  claim 48 , wherein the transduction compound is GABA (compound 20 in Table 1). 
     
     
         52 . The transduction buffer of  claim 48 , wherein the transduction buffer contains NDSB-201 and GABA as transduction compounds. 
     
     
         53 . The transduction buffer of  claim 48 , wherein the transduction buffer further comprises an osmoprotectant. 
     
     
         54 . The transduction buffer of  claim 53 , wherein the osmoprotectant is glycine and/or glycerol. 
     
     
         55 . The transduction buffer of  claim 53 , wherein the osmoprotectant is at a concentration of between about 5 mM and about 500 mM. 
     
     
         56 . The transduction buffer of  claim 48 , wherein the transduction buffer further comprises a biological pH buffer, a viscosity enhancer and/or a growth factor. 
     
     
         57 . The transduction buffer of  claim 48 , wherein the transduction compound is at a concentration of between about 0.1 mM and about 500 mM. 
     
     
         58 . The transduction buffer of  claim 48 , wherein the transduction compound is at a concentration of between about 200 mM and about 400 mM. 
     
     
         59 . The transduction buffer of  claim 48 , wherein the transduction buffer further comprises a molecule of interest for transduction, wherein the molecule of interest is a protein. 
     
     
         60 . The transduction buffer of  claim 59 , wherein the protein is a protein capable of modifying a nucleic acid. 
     
     
         61 . The transduction buffer of  claim 60 , wherein the protein capable of modifying a nucleic acid is a zinc finger nuclease, a TALEN, Cas9, a Cas9 analog, a DNA-targeted FokI-nuclease-associated protein, a Cascade complex, a TtAgo protein, another Argonaute protein, or their derivatives. 
     
     
         62 . The transduction buffer of  claim 60 , wherein the protein capable of modifying a nucleic acid is Cas9. 
     
     
         63 . A pharmaceutical composition comprising the transduction buffer of  claim 48 . 
     
     
         64 . The pharmaceutical composition of  claim 63 , wherein the pharmaceutical composition comprises a pharmaceutically acceptable carrier selected from water, saline, glycerol and ethanol.