Diseases that we discussed in some detail

 

Be able to discuss the following issues on each disease: What is the disease, i.e. how is it transmitted, what mutations/genes involved, what is the cellular/molecular effect of the disease, and anything else relevant. The scope of answers to these questions will be different for each disease. Make a study guide.

 

Alpha-1 antitrypsin deficiency

Myotonic dystrophy

Heritable cancers

Alzheimer’s disease

Alcohol dependence

 

 

Terms and concepts to know for the exam

 

This list of words from the textbook is intended to be a set of words whose definition you should know, but you should also be able to discuss the context in which each concept was presented in the book.

 

Human genome

Gene

Base pairs

Haplotype

Allele/variant/polymorphism/mutation/SNP

Genetic marker

RFLP

Linkage disequilibrium

Genetic linkage

Synteny

Heritability

Complex disease

Single-gene/monogenic/simplex disease

Mendelian inheritance  

Genetic risk

Molecular defect

Cellular defect 

Molecular mechanism

Mutational mechanism

Genetic map

Physical map

Centimorgan

Recombination

Concordance

Single sequence DNA

Intermediate repeated DNA

Highly repetitive DNA

Microsatellite

Trinucleotide repeat expansion (TRE)

Coding sequence

Intron

Exon

Promoter

Heterozygote

Homozygote

Genetic testing

Gene therapy

Monozygotic twins

Dizygotic twins

LOD score

Recombinant fraction

Recessive

Dominant

Autosomal

Sex-linked

Nuclear

Mitochondrial

Chromosomal dislocation

Nondisjunction

Hardy-Weinberg equilibrium

Phylogeny

Pathology

Etiology

Oncogene

Tumor suppressor

Congenital

Heritable cancer

Retinoblastoma

Complex disease

Simple disease

Polygenic

Parametric model

Non-parametric model

LOD score

Relative risk

Threshold of liability

Additive

Multiplicative

Linkage

Association

Genome scan

Sib-pairs

Unaffected sib-pairs

Discordant sib-pairs

Identical by descent (IBD)

Identical by state (IBS)

Homozygous

Heterozygous

Microsatellite markers

DNA sequence

Restriction fragment length polymorphism (RFLP)

VNTR (variable number tandem repeat)

Polymerase chain reaction (PCR)

Significant P value (0.05)

Linkage disequilibrium

Multipoint mapping

Autoimmune disorders

Transmission disequilibrium tests

IDDM, Type I diabetes

NIDDM, Type 2 diabetes

Insulin locus (INS)

Antigens

Twin concordance

Alzheimer's disease

Alcohol dependence/abuse

ADH (alcohol dehydrogenase)

ALDH (aldehyde dehydrogenase)

Genotype

Haplotype

Candidate gene

Gene therapy - somatic and germ line

Gene replacement vs. gene addition

in vivo vs ex vivo

Transgene

Cell type

SCID (severe combined immunodeficiency) and ADA (adenosine deaminase)

Clinical improvement

Animal models

Retrovirus

Adenovirus (AV)

AAV (adeno-associated virus)

Lentivirus

Vector

Heteroduplex

Virion

Genetic testing

Large-scale deletion

Small-scale deletion

Promoter mutation

Splice site mutation

Coding sequence

Non-coding sequence

Amino acid change

Protein-truncating mutations

Base substitutions

Tissue for testing

8-cell embryo

Chorionic villous sampling

Amniocentesis

Blood cells

Buccal cells

Multiplex PCR

Single-stranded conformational polymorphisms (SSCP)

Denaturing gradient gel electrophoresis (DGGE)

Mismatch cleavage detection

Protein truncation test

DNA chips/microarrays

DNA sequencing

Chromosome tracking

Denaturing high-performance liquid chromatography (DHPLC)

Neonatal screening

Preimplantation screening

Genetic counseling

Human Genome Project

Human diversity

Eugenics

Presymptomatic testing for predisposition to complex disease

Presymptomatic testing for late-onset disease

Genetic discrimination