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
DETECTING PROTEIN INTERACTION SITES IN NUCLEIC ACIDS
US Application 62/615,233
Filed on 2018-01-09
Filed on 2018-01-09
ISOTOPIC BIOMARKERS OF ORGANIC ACIDEMIAS
US Application 62/579,247
Filed on 2017-10-31
Filed on 2017-10-31
Biomarkers of Organic Acidemias
US Application 62/556,071
Filed on 2017-09-08
Filed on 2017-09-08
Novel Codon-optimized And Tagged Human NPC1 Genes And AAV Vectors For The Treatment Of Niemann-Pick Type C1 Deficiency And Related Conditions
US Application 62/522,677
Filed on 2017-06-20
Filed on 2017-06-20