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BMB 462: EXAM 2

What color topped vacutainer is used to collect blood for karyotyping? What is such a tube coated with.
GREEN Sodium heparin
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What is the active ingredient in prekaryotyping treatment?
colchicine
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acrocentric
centromere near end of chromosome
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metacentric
centromere near middle of chromosome
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submetacentric
centromere nearer to one end of chromosome than the other
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karyogram
picture of chromosomes
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karyotype
description of chromosome
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euploidy
cell with a set or sets of 23 chromosomes
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aneuploidy
chromosome number that is not an exact multiple of n (23 chromosomes)
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triploidy
double chromosome contribution from one parent
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DIANDRIC triploidy
extra set of chromosomes is paternal aka: TYPE I triploidy
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DIGYNIC triploidy
extra set of chromosomes is maternal aka: TYPE II triploidy (less common than type I)
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aneuploidy
cytogenetic abnormalities where all or part of one or more chromosomes is added or deleted
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aneuploidy is the result of ______
nondisjunction (failure of chromosomes to separate normally during cell division)
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viable autosomal trisomies
Down syndrome (trisomy 21) Patau syndrome (trisomy 13) Edward syndrome (trisomy 18)
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viable sex aneuploidies
Turner's syndrome (45, X) Kleinefelter syndrome (47, XXY) 47, XYY
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MONOsomy is ____ tolerated than TRIsomy
LESS
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Crossing over with PARAcentric inversion
NOT viable
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Corssing over with PERIcentric inversion
VIABLE
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Robertsonian translocations
two acrocentric chromosomes fusing at centromeres with loss of satellites and short arms - lost material (rRNA genes) is redundant phenotypically normal
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21q21q Transloaction (Robertsonian)
all surviving progeny will have trisomy 21
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isochromosomes
mirror image chromosomes two p arms or two q arms joined at the centromere
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Obligate carrier
individual who is assumed to be a carrier based on other affective relatives
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Consanguinity
parents share a common ancestor
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Dosage compensation achieved by ____
X-inactivation in females
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3 examples of X-lined recessive disorder
Hemophilia A Fragile X Syndrome Duchenne Muscular Dystrophy Red-green color blindness
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Example of X-linked dominant conditio
Rett syndrome
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Homoplasmy
all mitochondrial DNA are the same (either with or without a mutation)
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Heteroplasmy
proportion of normal and mutated mitchondrial DNA in a cell (proportion can change as cells divide)
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Clinical expression of mitochondrial inheritance depends on (3 things)
amount of heteroplasmy tissue distribution of mutatnt mtDNAs threshold effect (vulnerability of each tissue ot impaired oxidative metabolism)
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Key pattern for dominant inheritance
vertical transmission
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key pattery for recessive inheritance
skipping generations
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key pattern for X-linked recessive inheritance
bias towards affected males
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key pattern for x-lined dominant inheritance
bias towards affected females
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key pattern for mitochondrial diseases
affected mothers with all children affected
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What inheritance pattern rules out X-linked and mitochondrial disorders?
father-to-son transmission
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What inheritance pattern makes autosomal inheritance LESS LIKELY?
gender bias
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If males transmit the disease, you can rule out ____
mitochondrial inheritance
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If an unaffected mother has affected children, or if an affected mother has unaffected children, you can rule out _____
homoplasmic mitochondrial inheritance
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Penetrance
probability that an individual with a specific genotype will express a phenotype to any degree
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Complete penetrance
everyone with a given genotype will express the phenotype to some degree
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reduced penetrance
not everyone with a particular genotype will express the phenotype
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Example of reduced penetrance
Hereditary cancer syndrome, e.g.: retinoblastoma
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Causes of variability in phenotypic expression (6)
(1) reduced penetrance (2) variable expressivity (3) pleiotropy (4) allelic heterogeneity (5) locus heterogeneity (6) delayed age of onset
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Variable expressivity
manifestation of a disease is variable amongst individuals that have the same genotype
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Example of disease displaying variable expressivity
tuberous sclerosis
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Pleiotropy
a single gene producing diverse phenotypic effects; a pleiotropic ds gene could have multiple adverse effects in different organ systems/body areas
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Example of pleiotropic genetic diseases
Marfan's syndrome Cystic fibrosis
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Allelic heterogeneity
several different mutations of one gene that all cause the same disease
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Example of disease displaying allelic heterogeneity
cystic fibrosis
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Locus heterogenity
mutations at different loci (in different genes) causing the same disease or phenotype
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Example of locus heterogenieity
hearing loss
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Example of disease displaying delayed age of onset
Huntington ds
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Variability caused by interactions of genes (4 things)
(1) incomplete dominance (2) codominance (3) epistasis (4) sex-influenced conditions
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Incomplete dominance
expression of heterozygote (Aa) is different from and intermediate to both homozygotes (AA, aa)
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2 examples of disease showing incomplete dominance
achondroplasia familial hypercholesteremia
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Co-dominance
different alleles have an equal effect on the phenotype - both are expressed
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Example of co-dominance
ABO blood groups
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Epistasis
expression of one gene is prevented or modified by another gene (at a different locus)
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Example of epistasis
H gene codes for the H antigen on teh surface of RBCs
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Sex-influenced diseases are expressed ...
in both genders, but with significantly different frequencies
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Example of sex-influenced traits
male-pattern baldness (not an X-linked trait)
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Sex-limited condition
expressed exclusively in one gender
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Phenocopies
an environmental influence mimicking a genetic disorder
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Somatic mosaicism
mutation occurs AFTER fertilization, affecting a subpopulation of cells Individual manifests ds if percentage of affected cells is high enough
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Example of somatic mosaicism
segmental neurofibromatosis
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Germ line mosacisim
Subpopulation of gametes have mutation that individual does not himself express
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In germ line mosacisim, mutation occurs ___ fertilization in the process of ____, not ____
AFTER fertilization in the process of MITOSIS (not meiosis)
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2 examples of germ line mosaicism
DMD osteogenesis imperfecta
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Skewed X inactivation
one cell line (paternal X chr or maternal X chr) is preferentially inactiviated
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Hereditary hemochromatosis
increased iron absorption leads to multiple manifestations
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Mechanism for microdeletions
recombination error leading to duplication and deletion of chromosomes
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Wolf-Hirschhorn syndrome - genetic etiology
loss of terminal material from shot arm of chromosome 4
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Symptoms of Wolf-Hirschhorn syndrome
profound mental retardation seizures ocular hypertelorism broad or beaked nose micrognathia
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Cri-du-chat syndrome - genetic etiology
5p deletion
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Cri-du-chat syndrome: phenotype
newborns have weak, high pitched cat cry micrognathia microencephaly hypertelorism epicanthal folds low set ears slow growth severe developmental delay mental retardation
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VeloCardial Facial Syndrome (DiGeorge Syndrome) - genetic etiology
Chromosome 22q11 microdeletion
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VeloCardial Facial Syndrome - embryology
abnormalities affect 3rd and 4th branchial arches of embryonic development, leading to developmental abnormalities in parathyroid and thymus glands, and heat
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VeloCardial Facial Syndrome - phenotype
parathyroid hypoplasia - hypocalcemia thumic aplasia - immune deficiency conotruncal heart defects dysmorphic facial features cleft lip, cleft palate developmental delay increased risk for schizophrenia genital abnormalities
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William's syndrome - genetic etiology
Chromosome 7q11.23 microdeletion (elastin gene)
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Overarching theme of William's syndrome phenotype
Connective tissue disorder!
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Symptomes of William's syndrome
microcephaly and elfin-like facial features supravalvular aortic stenosis bladder diverticula hypercalcemia
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Inheritance pattern for Neurofibromatosis: Type 1 (NF1)
autosomal dominant inheritance (single gene mutation) with complete penetrance, but pleiotropy and variable expressivity 1/2 all new cases = de novo mutation no ethnic, geographical, sex, etc. bias
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Diagnostic criteria for NF1
six or more cafe au lait macules skinfold freckling 2+ neurofibromas optic glioma (w/ tunnel vision, HA, etc) 2+ Lisch nodules osseous lesion
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Pts w/ NF1 have a high risk for ___
CA - NF1 is an overgrowth syndrome!
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NF2 is due to mutations in what gene (nickname)?
Merlin gene
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Key phenotypic sign of NF2
bilatera CN8 vestibular schwannomas (symptoms of tinnitus, hearing loss and balance dysfunction)
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Penetrance of NF2
100% (complete penetrance), with variable expressivity and sometimes delayed age of onset
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Inheritance pattern for Marfan syndrom
Autosomal dominant with 100% penetrance
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Marfan syndrome caused by mutation to what gene
FBN-1 gene (fibrillin)
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Diagnostic criteria for Marfan's
skeletal manifestations cardiovascular optic dural ectasia family (NOT DNA testing)
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Three means of determining relative importance of genes vs. environment in ds etiology
Twin studies Concordance rates Heritability
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If concordance rates are similar, heritability is ____
LOW
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concardance rates
how often two individuals share the same trait/conditio
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traits mostly determined by genes will have a heritability close to ___
1.0
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heritability does NOT tell you anything about
how many genes could be involved how much each gene contributes to phenotypes mode of inheritance
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Genetic anticipation
aka: Sherman paradox increased frequency of affective relatives, severity of phenotype or earlier age of onset in successive generations
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Etiology of genetic anticipation phenomenon
trinucleotide expansion of a CGG repeat triplet sequence normally presents in the promoter of the FraX gene - greater number of repeats, greater chance individual is affected
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Premutation
expansion of a trinucleotide repeat, not significant enough to cause phenotype unstable - may expand to full mutation in next generation
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Is expansion usually maternal, paternal or both in FraX syndrome?
maternal
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threshold CGG repeat number for phenotype manifestation in FraX
200
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2 classes of trinucleotide repeat disorders
repeats in noncoding regions of a gene repeats in exonic region of gene
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2 clinical entities relative to FMR-1 locus
premature ovarian failure FraX-associated Tremor/Ataxia Syndrome
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genetic description of Myotonic dystrophy
CTG expansion in 3' UTR of DMPK gene (codes for a myotonin-protein kinase)
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protein-level problem with DTG expansion in 3' UTR of DMPK gene
protein levels unaffected problem apparently with expanded mRNA and its processing
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CTG premutation repeat can ___ after MATERNAL transmission and ____ after PATERNAL transmission
maternal: expand paternal: contract
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In Huntington's ds, expansion preimary occurs after ___ inheritance
paternal
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define polyglutamine diseases
CAG repeats (CAG = dodon for glutamine)
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polyglutamine causes ____ in the ____
polyglutamine causes protein aggregation in the nucleus
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CAG repeats in HD are considered __-of-function mutations
GAIN-of-function
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GENETIC IMPRINTING
different expression of genetic material, at either the chromosomal or allelic level, depending on whether the geneitc materal is inherited from the male or female parent
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5 disease indicating genetic imprinting
HD myotonic dystrophy Fragile X autosomal dominant spinocerebellar ataxia NF1
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hydatidiform mole
occurs when chromosomes in fertilized egg are mostly or all paternal in origin trophoblastic proliferation with little to no embyronic development
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Imprinting pattern of an individual is established ____ fertilization and ____ during life
Imprinting pattern of an individual is esatblished BEFORE fertilization and remains established during life.
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What biochemical phenonemenon is strongly associated with imprinting
DNA methylation
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2 clinical manifestations of imprinting
Prader-Willi syndrome Angelman syndrome
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chromosome involved in Prader-Willi and Angelman syndromes
Chromosome 15 (q11-q13)
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Symptoms of Prader-Willi
neonatal hypotonia (similar to Trisomy 21) poor feeding, FTT to age 2 at age 2, hyperphagia short stature, small hands/feet, micropenis, hypogonadism mod to severe microencephaly obesity
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Symptoms of Angelman syndrome
usu. normal at birth, then manifest: progressive microcephaly severe MR, lack of speech onset gait ataxia abnormal EEF, seizures
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Deletions in Chr 15q11 inherited from MOTHER results in ____
Angelman syndrome
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Uniparental disomy - probable mechanism
initially trisomic zygote subsequently loses chromosome
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Equation for ALLELE frequencies
p + q = 1
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Equaltion for GENOTYPE frequencies
p^2 + 2pq + q^2 = 1
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Hardy-Weinberg Equilibrium Principle
Allele and phenotype frequencies remain constant in subsequent generations
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4 assumptions of H-W Equil.
Two allels random mating large population no migration, new mutations, natural selection
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Do H-W practice problems, p. 268
do them!
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2 types of enzymes required for recombinant DNA technology
Restriction endonucleases DNA ligases
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Biological function for restriction enzymes
bacterial defense mechanisms - cut up foreign DNA
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recombinant DNA
DNA molecule consisteing of components from different "parent" molecules
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cloning
making many copies of the recombinant DNA using a host organism
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vector
DNA molecule that can be replicated (i.e.: has an ORI specific to a particular host organism - bac, yeast, etc.)
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Insert
DNA fragment from organism/individual of interest, which will be joined to a vector
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host
organism that will replicate the recombinant DNA
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transformation
process by which teh recombinant DNA is put into the host
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2 essential features of vectors
insertion site origin of replication
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plasmid vectors
small, circular DNA vectors
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cDNA
capture just one protein coding region per ONE fragment (one insert) for cloning
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how to make cDNA
synthesize from mRNAs using reverse transcriptases
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what will cDNA not have?
introns promoters enhancers spacer DNA
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expression clones
special case of cDNA inserts get host cell to synthesize protein encoded by cDNA require expression vectors use bacterial RNA polymerase and bacteral ribosome to make eukaryotic protein
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clone libraries
collection of clones, made simultaneously, from a given sample
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How do you differentiate between autosomal dominant and x-linked dominant?
Can males lead to affected males? AD = Yes XD = No
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