ШЕФФЕР Дэвид В. (US),КЛИМЧАК Райан Р. (US),КЁРБЕР Джеймс Т. (US),ФЛЭННЕРИ Джон Дж. (US),ДАЛКАРА МОУРОТ Дениз (FR),ВИЗЕЛЬ Майке (US),БЁРН Леа С.Т. (US)
申请号：
RU2013151885/10
公开号：
RU2013151885A
申请日:
2012.04.20
申请国别(地区):
RU
年份:
2015
代理人:
摘要：
1. A recombinant virus virion (rAAV), comprising: a) an AAV variant capsid protein, characterized in that the AAV variant capsid protein contains an insert of about 5 amino acids to about 11 amino acids in the GH loop capsid protein compared to the corresponding parent AAV capsid protein, and where the variant capsid protein gives an increased infectivity of the retinal cell compared to the infectivity of the retinal cell with an AAV virion containing the corresponding parent AAV capsid protein； and b) a heterologous nucleic acid containing a nucleotide sequence encoding a gene product. Virion rAAV according to claim 1, characterized in that the insert is a peptide of Formula I, Formula IIa, Formula III or Formula IV.3. Virion rAAV according to claim 2, characterized in that the insert contains an amino acid sequence selected from LGETTRP (SEQ ID No. 13), NETITRP (SEQ ID No. 14), KAGQANN (SEQ ID No. 15), KDPKTTN (SEQ ID No. 16), KDTDTTR (SEQ ID No. 57), RAGGSVG (SEQ ID No. 58), AVDTTKF (SEQ ID No. 59) and STGKVPN (SEQ ID No. 60) .4. The rAAV virion according to claim 1, characterized in that the retinal cell is a photoreceptor, retinal ganglion cell, Mueller cell, bipolar cell, amacrine cell, horizontal cell or retinal pigment epithelial cell. The rAAV virion according to claim 1, characterized in that the insertion site is between amino acids 587 and 588 from AAV2, between amino acids 590 and 591 from AAV1, between amino acids 575 and 576 from AAV5, between amino acids 590 and 591 from AAV6, between amino acids 589 and 590 from AAV7, between amino acids 590 and 591 from AAV8, between amino acids 588 and 589 from AAV9, or between amino acids 588 and 589 from AAV10.6. The rAAV virion according to claim 1, characterized in that the rAAV virion exhibits at least 10-fold1. Вирион рекомбинантного вируса (rAAV), содержащий:a) вариантный капсидный белок AAV, отличающийся тем, что вариантный капсидный белок AAV содержит вставку из приблизительно 5 аминокислот до приблизител
