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Genetics > CRISPR/Cas9 > Flashcards

CRISPR/Cas9 Flashcards

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Biology 101 flashcards
1
Q

What is genome editing?

A

The intentional modification of an organism’s DNA at a specific location

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2
Q

What systems are powerful tools for genome editing?

A

CRISPR/Cas

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3
Q

What does CRISPR/Cas enable?

A

High efficacy targeting of nearly any site in the genome

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4
Q

What is the main Cas enzyme that people use?

A

Cas9 - Streptococcus pyogenes

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5
Q

What does Cas9 do exactly?

A

It complexes with RNAs that enable it to localize to specific locations in a genome and it has domains in it that can cleave nucleic acids

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6
Q

How do you program Cas9?

A

Program it with appropriate guide RNA and it will bind to a specific part of the genome and cleave a double strand break (DSB)

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7
Q

2 ways DSBs can be repaired?

A

With a donor DNA template
Nonhomolgous End Joining

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8
Q

What are the 3 types of Nonhomolgous End Joining

A

It can randomly:
Do a small insertion
Substitution
Small deletion

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9
Q

Basically put what CRISPR/Cas9 is for

A

You produce a double strand break in a location of the genome you want to change and then repair those breaks with genetic changes…
Can be used to fix diseases

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10
Q

What in Cas9 designates where it will go in the genome?

A

The protospacer

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11
Q

What is a PAM and why is it required?
What is the sequence?

A

Protospacer adjacent motif
It helps signal where cas9 can attach
“N”GG
N= any nucleotide

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12
Q

Who were the three principal investigators whose labs figured out how to use CRISPR/Cas systems to edit genomes?

A

Jennifer Doudna - Intellia
Emmanuelle Charpentier - CRISPR Theraputics
Feng Zhang - Editas

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13
Q

Name the three companies that are the first to attempt CRISPR/Cas-based genome editing therapies in humans

A

Editas, LCA10, in vivo
Intellia, ATTR amyloidosis, in vivo
CRISPR Theraputics, Sickle cell and B-thalessemia, ex vivo

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14
Q

Who started Editas?

A

Feng Zhang

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15
Q

What genetic disorder did Editas initially target?

A

EDIT 101: LCA10, in vivo
Also, EDIT-301 SCD

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16
Q

What is LCA and SCD?

A

Leber congenital amaurosis
and sickle cell disease

17
Q

What is LCA10?

A

it is the most common form of Leber congenital amaurosis

18
Q

What are 2 factors that contribute to EDIT 101 efficacy?

A

The retina is accesible to injection and since rods and cones do not divide, there is low risk for triggering genome instability and cancer.

19
Q

What diseases does CRISPR Theraputics target?

A

Sickle Cell Disease and Beta-Thalassemia

20
Q

What is the CRISPR Tx program that targets SCD and B-Thalass.

A

CTX001 / Exa-cel

21
Q

What does CTX001/Exa-cel do at the molecular level?

A

It turns the fetal hemoglobin gene (HbF) back on to represses broken adult hemoglobin gene (HbA)

22
Q

What disease does Intellia Theraputics target?

A

ATTR Amylodiosis

23
Q

What is ATTR Amyloidosis?

A

Accumulation of misfolded TTR which affects nerves, heart, kidneys and eyes.

24
Q

What program at Intellia targets ATTR Amyloidosis?

A

NTLA-2001

25
Q

What does NTLA-2001 do?

A

Break the TTR gene to reduce TTR levels

26
Q

What are concerns for editing people’s cells?

A

Triggering genome instability leading to cancer
Being able to deliver reagents to the appropriate part of the body
Ethics- editing in the U.S. is on somatic cells, no embryos or germline

27
Q

What are CAR-T cells?

A

Chimeric Antigen Receptor cells are T cells genetically engineered to target cancer cells

28
Q

A major CRISPR/Cas objective is to make allogenic CAR-T cells, what does allogenic mean?

A

Cells that can be used by any person

29
Q

Autologous vs Allogeneic?

A

Autologous - Cells derived from the cancer patient used to produce one treatment
Allogenic - cells derived from a healthy donor and manufacturing ready to use CAR-T cells, saving time and more treatments as needed

30
Q

What are 4 other uses for CRISPR/Cas?

A
  1. Therapies based on controlling transcription at one or more genes.
  2. Antiviral/Antipathogen therapies
  3. Crop productivity across climates
  4. Diagnostics

Genetics (17 decks)

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