Cystic Fibrosis

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Genetic Abnormalities  How they occur  Sample Collection of Genetic Disorders Human Genome Project Information Website •Human Genome Project Information •Gene Gateway •Research •Education •Medicine •Ethical, Legal & Social Issues Human Genome Project Information Genes and Disease  National Center for Biotechnology Information (NCBI)   A division of the National Library of Medicine at the National Institute of Health Research & collaborative data collection and storage for researchers to better understand the link to genetics with health and disease Chromosome Map Genetic Testing  Genetic Counseling  Pedigree Analysis  Blood test for proteins  Phenotype analysis  Fetal Testing  Karyotype  Amniocentesis  Chorionic villus sampling (CVS)  Newborn Testing Chromosomal Errors: Problems in Meiosis  Nondisjunction:  Aneuploidy: • Monosomy • Trisomy • Polyploidy Down’s Syndrome  Extra chromosome 21 (autosomal)  Trisomy, from nondisjuntion  Short stature; characteristic facial features; mental ability is variable; often problems with the circulatory system Figure 5.15 Klinefelter’s Syndrome  Extra sex chromosome (XXY)  Trisomy,from nondisjuntion  Infertile male (extra X interferes with meiosis); feminine physical traits; below average intelligence; taller than average Turner’s Syndrome  One less sex chromosome (XO)  Monosomy, resulting from nondisjuntion  Infertile female; sex organs don’t fully develop  Typically below ave. height; normal intelligence Chromosomal errors: Alterations of chromosomal structure Deletion Inversion Base Substitution Duplication Translocation Repeats Cystic Fibrosis  3 base-pair deletion in sequence on chromosome 7  Autosomal Recessive Inheritance  Produces a defective chloride ion membrane protein  an increase of salt (NaCl) in bodily secretions  body produces a thick, sticky mucus  that clogs the lungs causing infection  blocks the pancreas stopping digestive enzymes from reaching the intestines for food digestion Phenylketonuria (PKU)  Located on chromosome 12  Autosomal Recessive Inheritance  Failure to convert phenylalanine to tyrosine (lacks phenylalanine hydrolyase) toxic levels in the blood mental retardation  Treatment- avoidance of foods with phenylalanine Albinism  Located on chromosome 11- deletion  Autosomal Recessive Inheritance  Tyrosinase deficiency  Blocks pathway to produce melanin Sickle Cell Anemia  Located on chromosome 11  Base substitution  single amino acid substitution  Autosomal Recessive Inheritance  Produces mutated form of hemoglobin  Blocks capillaries localized hypoxemia Achondroplasia  Located on chromosome 4  Autosomal Dominant Inheritance  Homozygous dominant is lethal disease  Form of dwarfism Figure 14.15 Huntington Disease  Located on chromosome 4  Repeat  Autosomal Dominant Inheritance  Build up of toxic proteins on the brain nervous system breaks down  Adult onset, usually during or after reproductive years Red-Green Colorblindness  Located on X chromosome  Deletions due to unequal crossing over  Sex-linked Recessive Inheritance  A range of phenotypes, depending upon the type of deletion http://www.thetech.org/genetics/images/ask/colorblind.jpg http://www.coolopticalillusions.com/optical_illusions_pictures_3/color_blindness_test.htm Hemophilia  Located on X chromosome  Base substitution  Sex-linked Recessive Inheritance  Inability to make one/more of the clotting factors  bruising, prolonged bleeding, anemia Duchenne’s Muscular Dystrophy  Located on X chromosome- large gene high mutation rate  Sex-linked Recessive Inheritance  Makes defective protein, causing muscle to weaken Cri-du-Chat  Deletion of half of chromosome 5  “cry of the cat”- characteristic of infants  Mental retardation; slow growth; small head; finger webbing Cri-du-Chat YouTube video Fragile X Syndrome  Sex-linked Dominant Inheritance  repeat sequence CGG     Normal repeats (Variable Number Tandem Repeat- VNTR) = 5-50 Premutation = 50-200 repeats Full Mutation = over 230 repeats!! Leads to FMR1 gene not expressed- no FMRP  Plays a role in synaptic connections of nerve cells  Not always expressed due to x-inactivation  Most common cause of mental retardation  Distinctive physical appearance  Long face, large ears Angelman & Prader Willi Syndromes  Deletions on chromosome 15  Genomic imprinting  If from father- Prader Willi Syndrome  characterized by obesity low muscle tone, cognitive disabilities, & incomplete development severe mental retardation development, speech, movement)  If from mother- Angelman Syndrome   unusual facial appearance  apparent happy demeanor  muscular abnormalities  hand flapping Angelman vs Prader Willi Syndromes

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