ABERRANT POST-TRANSLATIONAL MODIFICATIONS (PTMS) IN METHYL- AND PROPIONIC ACIDEMIA AND A MUTANT SIRTUIN (SIRT) TO METABOLIZE PTMS Read more about ABERRANT POST-TRANSLATIONAL MODIFICATIONS (PTMS) IN METHYL- AND PROPIONIC ACIDEMIA AND A MUTANT SIRTUIN (SIRT) TO METABOLIZE PTMS US Application 63/021,179Filed on 2020-05-07
Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In Propionyl-coA Carboxylase Alpha (PCCB) Read more about Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In Propionyl-coA Carboxylase Alpha (PCCB) US Application 62/915,347Filed on 2019-10-15
Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In PropionylcoA Carbox Lase Alpha (PCCA) Read more about Synthetic Genes For The Treatment Propionic Acidemia (PA) Caused By Mutations In PropionylcoA Carbox Lase Alpha (PCCA) US Application 62/867,374Filed 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 Read more about Targeted Integration Into The Albumin (Alb) Locus As A Treatment For Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MUT) Deficiency US Application 62/177,771Filed on 2018-08-10
DETECTING PROTEIN INTERACTION SITES IN NUCLEIC ACIDS Read more about DETECTING PROTEIN INTERACTION SITES IN NUCLEIC ACIDS US Application 62/615,233Filed on 2018-01-09
ISOTOPIC BIOMARKERS OF ORGANIC ACIDEMIAS Read more about ISOTOPIC BIOMARKERS OF ORGANIC ACIDEMIAS US Application 62/579,247Filed on 2017-10-31
Biomarkers of Organic Acidemias Read more about Biomarkers of Organic Acidemias US Application 62/556,071Filed 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 Read more about 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 62/522,677Filed on 2017-06-20
METHODS OF DETECTING AND TREATING GATM-RELATED RENAL DISORDERS Read more about METHODS OF DETECTING AND TREATING GATM-RELATED RENAL DISORDERS US Application 62/515,834Filed on 2017-06-06
CANCER DETECTION AND CLASSIFICATION Read more about CANCER DETECTION AND CLASSIFICATION US Application 62/898,670Filed on 2019-09-11