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  2. Lysosomal storage disease - Wikipedia

    en.wikipedia.org/wiki/Lysosomal_storage_disease

    Tay–Sachs disease was the first of these disorders to be described, in 1881, followed by Gaucher disease in 1882. In the late 1950s and early 1960s, de Duve and colleagues, using cell fractionation techniques, cytological studies, and biochemical analyses, identified and characterized the lysosome as a cellular organelle responsible for ...

  3. Mitochondrial disease - Wikipedia

    en.wikipedia.org/wiki/Mitochondrial_disease

    Mitochondrial disease is a group of disorders caused by mitochondrial dysfunction. Mitochondria are the organelles that generate energy for the cell and are found in every cell of the human body except red blood cells. They convert the energy of food molecules into the ATP that powers most cell functions.

  4. Mucopolysaccharidosis - Wikipedia

    en.wikipedia.org/wiki/Mucopolysaccharidosis

    Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans (GAGs). These long chains of sugar carbohydrates occur within the cells that help build bone, cartilage, tendons, corneas, skin and connective tissue.

  5. Ribosomopathy - Wikipedia

    en.wikipedia.org/wiki/Ribosomopathy

    In humans, as in most eukaryotes, the 18S rRNA is a component of 40S ribosomal subunit, and the 60S large subunit contains three rRNA species (the 5S, 5.8S and 28S in mammals, 25S in plants). 60S rRNA acts as a ribozyme, catalyzing peptide bond formation, while 40S monitors the complementarity between tRNA anticodon and mRNA. [citation needed]

  6. Pearson syndrome - Wikipedia

    en.wikipedia.org/wiki/Pearson_syndrome

    Pearson syndrome is a mitochondrial disease caused by a deletion in mitochondrial DNA (mtDNA). [3] An mtDNA is genetic material contained in the cellular organelle called the mitochondria. Depending on the tissue type, each cell contains hundreds to thousands of mitochondria. There are 2–10 mtDNA molecules in each mitochondrion.

  7. Hurler syndrome - Wikipedia

    en.wikipedia.org/wiki/Hurler_syndrome

    As of 2018, more than 201 different mutations in the IDUA gene have been shown to cause MPS I. [6] Because Hurler syndrome is an autosomal recessive disorder, affected persons have two nonworking copies of the gene. A person born with one normal copy and one defective copy is called a carrier. They will produce less α-L-iduronidase than an ...

  8. Gaucher's disease - Wikipedia

    en.wikipedia.org/wiki/Gaucher's_disease

    The disease is caused by a defect in the housekeeping gene for lysosomal glucocerebrosidase (also known as beta-glucosidase, EC 3.2.1.45 ) on the first chromosome (1q22). The enzyme is a 55.6- kilodalton , 497- amino acid -long protein that catalyses the breakdown of glucocerebroside, a cell membrane constituent of red and white blood cells .

  9. Glycogen storage disease type II - Wikipedia

    en.wikipedia.org/wiki/Glycogen_storage_disease...

    Glycogen storage disease type II has an autosomal recessive pattern of inheritance. Pompe disease has an autosomal recessive inheritance pattern. This means the defective gene is located on an autosome, and two faulty copies of the gene—one from each parent—are required to be born with the disorder. As with all cases of autosomal recessive ...