US 12,214,055 B2
Systems and methods for treating hyper-igm syndrome
Cecillia Cotta-Ramusino, Cambridge, MA (US); Carrie M. Margulies, Waban, MA (US); Luigi Naldini, Milan (IT); and Pietro Genovese, Milan (IT)
Appl. No. 16/758,752
Filed by EDITAS MEDICINE, INC., Cambridge, MA (US); FONDAZIONE TELETHON ETS, Rome (IT); and OSPEDALE SAN RAFFAELE, Milan (IT)
PCT Filed Oct. 24, 2018, PCT No. PCT/US2018/057354
§ 371(c)(1), (2) Date Apr. 23, 2020,
PCT Pub. No. WO2019/084168, PCT Pub. Date May 2, 2019.
Claims priority of provisional application 62/746,900, filed on Oct. 17, 2018.
Claims priority of provisional application 62/690,284, filed on Jun. 26, 2018.
Claims priority of provisional application 62/673,039, filed on May 17, 2018.
Claims priority of provisional application 62/664,800, filed on Apr. 30, 2018.
Claims priority of provisional application 62/576,277, filed on Oct. 24, 2017.
Prior Publication US 2020/0338213 A1, Oct. 29, 2020
Int. Cl. A61K 48/00 (2006.01); A61K 39/00 (2006.01); A61P 37/02 (2006.01); C07K 14/475 (2006.01); C07K 14/705 (2006.01); C12N 15/10 (2006.01); C12N 15/113 (2010.01); C12N 15/86 (2006.01); C12N 15/90 (2006.01); A61K 35/17 (2015.01)
CPC A61K 48/005 (2013.01) [A61K 39/461 (2023.05); A61K 39/4611 (2023.05); A61K 39/464411 (2023.05); A61P 37/02 (2018.01); C07K 14/475 (2013.01); C07K 14/70575 (2013.01); C07K 14/70578 (2013.01); C12N 15/102 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 15/907 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/50 (2013.01); C12N 2840/203 (2013.01)] 20 Claims
 
1. An isolated oligonucleotide donor template which comprises, from 5′ to 3′, A1-N-UTR-pA-A2, wherein
A1 is a homology arm that is substantially identical to a first homology arm of a target CD40L nucleic acid;
N is a cargo comprising one or more of exons 2-5 of a CD40L gene;
UTR is a CD40L 3′ untranslated region (UTR);
pA is a polyA tail; and
A2 is a homology arm that is substantially identical to a second homology arm of the target CD40L nucleic acid,
and wherein the cargo does not comprise exon 1 of CD40L.