More examples in PM Flashcards

1
Q

How does PCSK9 work?

A

PCSK9 is a moleulce that leads to the regulation of LDL in the blood. The issue with it is that it causes receptor and LDL to remain together and both are sent to hte lysosome for degredation.

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2
Q

What are the two PCSK9 inhibitors we should know?

A

Alirocumab
Evolocumab

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3
Q

How much does the LDL cholesterol decrease by with PCSK9 inhibitors onboarded?

A

55-60%

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4
Q

What is VCTX210?

A

Allogeneic gene edited stem cell derivted therapy that is used for individuals who have T1D and insulin dependent T2D.

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5
Q

What is special about VCTX210?

A

Engineered to evade the immune resposne for increased persistence which also eliminates the immune suppression therapy

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6
Q

How does VCTX210 work?

A

Engineered to to repalce the beta cells that are lost in diabetes and is developed using CRISPR gene editing appraoch

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7
Q

What is ViaCyte?

A

Cell replacement therapy that is an implant under the ksin of the patient and can mature into beta cells and other islet cells.

Immune based rejection can occur though

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8
Q

How can we avoid the immune activation of T cell activation in Viacute?

A

Crispr is utilized to KO B2M which is important for MHC/LA, therefore T cell is not activated when it touches the cells.

CRISPR is also used to add PD-L1 which further suppresses T cell activation

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9
Q

What are the two biomarkers for alzheimers?

A

TAU tangles and Amyloid plaques

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10
Q

What happens when we have the development of amyloid plaques and TAU tangles?

A

and the Amyloid plaques form cluster blocks between cells

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11
Q

What is Aducanumab?

A

This is a MAB for the treatment of alzheimers disease that delays the loss of function for early stage of the disease , it works by binding to Amyloid plaques and it causes it to bind to microglia to then digest the large extracellular particles

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12
Q

What is KarXT therapy for?

A

The goal is for the treatment of schizophrenia.

It is a combo medicaiton where one medicaiton Increases activity in all regions such as CNS, Salivaton, Sweat glands etc… while the other medication counteracts the other side effects. Therefore leading to a neutralizing effect.

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13
Q

With KarXT therapy how does it work?

A

2 medications. One increases actiivty and the other decreases activity.

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14
Q

What are neoantigents?

A

They are specific mutations that are not on normal cells that we can target.

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15
Q

How are mRNA based vaccines for cancner working?

A

Using neoantigens. We can then really target someones specific cancer treatment to them!

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