DNA and Games
The genome is the total ______ in an organism.
What is Deoxyribonucleic acid (DNA)
Gene therapy involves replacing, manipulating, or supplementing ________ genes with healthy genes into target cells to treat patients.
What are nonfunctional (malfunctioning) genes?
The procedure awarded the Nobel prize in Physiology or Medicine in 2007 to Drs. Mario R. Capecchi, Martin J. Evans, and Oliver Smithies for introducing specific gene modifications in mice involve the use of what cell type for gene targeting?
What are Embryonic stem cells?
This repair pathway involves the exchange between homologous chromosomes or a donor template.
What is homology directed repair or homologous recombination?
This early gene editing tool has a protein binding domain consisting of eukaryotic transcription factors containing zinc finger motifs.
What are Zinc finger nucleases?
This short sequence is necessary for selectively shuttling Cas9 proteins into the nucleus.
What is the nuclear localization signal (NLS).
The first step in central dogma.
What is Transcription?
This is the definition of a transgene.
What is the expression of a healthy gene?
The procedure awarded the Nobel prize in Physiology or Medicine in 2007 to Drs. Mario R. Capecchi, Martin J. Evans, and Oliver Smithies for introducing specific gene modifications involves this type of DNA repair pathway?
What is Homologous recombination?
This phenomena represents the fundamental basis for gene editing using nucleases, such as ZFNs and CRISPR-Cas9.
What are double-strand breaks?
In ZFNs, each zinc finger domain recognizes this number bases of sequence.
What are 3 bases?
CRISPR-Cas9 was adapted from what type of organisms and performed which function?
What is the bacterial adaptive immune system?
The transfer of a mutation in any of the numbered chromosomes is referred to as what type of inheritance pattern?
What is Autosomal?
What type of gene therapy has been banned globally use to ethical issues?
What is Germline gene therapy?
This duo of female scientist were awarded the Nobel Prize in Chemistry for CRISPR-Cas9?
Who are Jennifer Doudna and Emmanuelle Charpentier?
This DNA repair pathway is independent of a template or homologous DNA sequence, but can involve random insertions or deletions at the break site.
What is Non-homologous end joining?
For protein-based gene editing tools, ZFNs and TALENs, you need a pair of nucleases to bind to separate DNA strands in the target locus with sufficient spacing to accommodate this next step. Note that this step is required for DSB to occur.
What is FokI dimerization?
The CRISPR array encode for alternating repeats and _______.
What are spacers?
An inheritance pattern where you have the disease phenotype in the presence of one copy of the diseased allele is referred to as what inheritance mode?
What is dominant mode of inheritance?
These are obligate intracellular parasites require a host to replicate?
What discovery simplified the CRISPR-Cas9 technology for editing in any cell type?
What are chimeric sgRNA?
This pathway also known as alternative non-homologous end-joining involves single strand annealing of 30 bp or more of complementary sequence and is a source of large deletions.
What is microhomology-mediated end-joining?
This solution to the ZFN design is used to mitigate off-target gene editing?
What are obligate heterodimers (consisting of mutant FokI proteins)?
The CRISPR array is transcribed into this RNA.
What are precursor CRISPR RNA (pre-crRNA)?
Provide the term that describes the state of being heterozygous for a mutation in different cell types?
What is genetic mosaicism?
This single stranded DNA virus is the delivery platform of choice for gene therapies and has been approved in recent products, such as Luxturna for retinal dystrophy?
What are Adeno-associated viruses (AAVs)?
Shinya Yamanaka and John Gurdon are stem-cell researchers who won the Nobel Prize in Physiology or Medicine in 2012 for what discovery?
What are Induced pluripotent stem cell (iPSC) technology?
This repair pathway is leveraged for making knockout models or disrupt a therapeutic target gene.
What is Non-homologous end joining (NHEJ)?
The first in human use of gene editing was for this devastating disease?
What is human immunodeficiency virus (HIV)?
This sequence must be present proximal to the target in order for the CRISPR-Cas9 to bind and activate a DSB.
What is protospacer adjacent motif (PAM)?