METHODS FOR TREATMENT OF SUBJECTS WITH PREEXISTING IMMUNITY TO VIRAL TRANSFER VECTORS
US Application 62/624,103
Filed on 2019-10-21
Filed on 2019-10-21
METHODS FOR TREATMENT OF SUBJECTS WITH PREEXISTING IMMUNITY
US Application 62/829,771
Filed on 2019-04-28
Filed on 2019-04-28
METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS
US Application 62/839,761
Filed on 2019-04-28
Filed on 2019-04-28
Novel Adeno-associated Viral (AAV) Vectors To Treat Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MMUT) Deficiency
US Application 63/080,337
Filed on 2020-09-18
Filed on 2020-09-18
METHODS AND COMPOSITIONS OF MMA CONSTRUCTS AND VECTORS
US Application 62/698,528
Filed on 2018-07-16
Filed on 2018-07-16
DIAGNOSIS AND TREATMENT OF VEXAS, WITH MOSAIC MISSENSE MUTATIONS IN UBA1
US Application 63/059,107
Filed on 2020-07-30
Filed on 2020-07-30
ABERRANT POST-TRANSLATIONAL MODIFICATIONS (PTMS) IN METHYL- AND PROPIONIC ACIDEMIA AND A MUTANT SIRTUIN (SIRT) TO METABOLIZE PTMS
US Application 63/021,179
Filed on 2020-05-07
Filed on 2020-05-07
Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In Propionyl-coA Carboxylase Alpha (PCCB)
US Application 62/915,347
Filed on 2019-10-15
Filed on 2019-10-15
Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In PropionylcoA Carbox Lase Alpha (PCCA)
US Application 62/867,374
Filed on 2019-06-27
Filed on 2019-06-27
Targeted Integration Into The Albumin (Alb) Locus As A Treatment For Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MUT) Deficiency
US Application 62/177,771
Filed on 2018-08-10
Filed on 2018-08-10