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Showing 1 to 4 of 4 for “"inborn error of metabolism"”.

  1. Consequences and Prevention of Elevated Circulating Tyrosine during Nitisinone Therapy in Alkaptonuria.

    … is an ultra-rare, autosomal recessive disorder of tyrosine catabolism due to mutations within the homogentisate 1,2-dioxygenase (HGD) gene. The resulting enzyme deficiency leads to accumulation of homogentisic acid (HGA) and deposition of melanin-like pigment polymers in the connective tissues …

    liverpool-jm Repository record for Consequences and Prevention of Elevated Circulating Tyrosine during Nitisinone Therapy in Alkaptonuria. (opens in a new tab)

  2. Generation of a Medaka Fish Model of Propionic Acidemia for Development of Novel Therapies.

    Propionic acidemia (PA) is an autosomal recessive inborn error of metabolism caused by deficiency of the mitochondrial enzyme propionyl-CoA carboxylase (PCC). The disease presents with acute, recurrent and life-threatening crises of metabolic decompensation starting from the newborn period. …

    the-open-u Repository record for Generation of a Medaka Fish Model of Propionic Acidemia for Development of Novel Therapies. (opens in a new tab)

  3. Structural investigations of adenosylcobalamin-dependent enzyme maturation

    Metalloenzymes utilize metallocofactors, ranging from single metal ions to complicated metallic clusters, to catalyze a wide range of challenging chemical reactions that are critical for life. Incorporation of these metallocofactors often relies on proteins known as metallochaperones that …

    mit Repository record for Structural investigations of adenosylcobalamin-dependent enzyme maturation (opens in a new tab)

  4. Isovalerianazidämie : Klinik, Pathobiochemie und Therapie am Beispiel von 28 Patienten

    Die Isovalerianazidämie ist eine Stoffwechselstörung im Abbau der verzweigtkettigen Aminosäure Leucin, der ein Defekt des Enzyms Isovaleryl-CoA-Dehydrogenase zugrunde liegt. In der vorliegenden Arbeit wurden 28 Patienten (15 männlich, 13 weiblich) mit Isovalerianazidämie bezüglich der Diagnostik, …

    freiburg-diss Repository record for Isovalerianazidämie : Klinik, Pathobiochemie und Therapie am Beispiel von 28 Patienten (opens in a new tab)