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Deciphering novel TCF4-driven mechanisms underlying a common triplet repeat expansion-mediated disease
by
Bhattacharyya, Nihar
,
Chai, Niuzheng
,
Hafford-Tear, Nathaniel J
,
Sadan, Amanda N
,
Szabo, Anita
,
Zarouchlioti, Christina
,
Jedlickova, Jana
,
Leung, Szi Kay
,
Liao, Tianyi
,
Dudakova, Lubica
,
Skalicka, Pavlina
,
Parekh, Mohit
,
Moghul, Ismail
,
Jeffries, Aaron R
,
Cheetham, Michael E
,
Muthusamy, Kirithika
,
Hardcastle, Alison J
,
Pontikos, Nikolas
,
Liskova, Petra
,
Tuft, Stephen J
,
Davidson, Alice E
Published in
PLoS genetics
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Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4
by
Sai, Hali
,
Ollington, Bethany
,
Rezek, Farah O.
,
Chai, Niuzheng
,
Lane, Amelia
,
Georgiadis, Anastasios
,
Bainbridge, James
,
Michaelides, Michel
,
Sacristan-Reviriego, Almudena
,
Perdigão, Pedro R.L.
,
Leung, Amy
,
van der Spuy, Jacqueline
Published in
Molecular therapy. Nucleic acids
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