Summary
Summary lectures Molecular therapy
- Course
- Institution
All the lectures of molecular therapy organised and summarized, good for study the final exam
[Show more]
Seller
Follow
mireiasabater24
Content preview
Molecular TherapyMOLECULAR THERAPY
KW1 Radboud Universiteit
2024/2025
0
,Molecular Therapy
1
,Molecular Therapy
TABLE OF CONTENTS
BLOC I: DRUG DELIVERY AND DEVELOPMENT ................................................................ 3
UNIT 1: Drug delivery .................................................................................................. 3
UNIT 2: Drug development ........................................................................................ 18
BLOC II: PHARMACODYNAMICS AND -KINETICS........................................................... 23
UNIT 3: Pharmacology and drug disposition .............................................................. 23
UNIT 4: Biotransformation ........................................................................................ 29
UNIT 5: Transporters ................................................................................................. 34
BLOC III: GENETIC THERAPY FOR RETINAL DISEASE ..................................................... 39
UNIT 6: Genetic therapy for inherited retinal diseases ................................................ 39
UNIT 7: Gene augmentation therapy (adding DNA) ..................................................... 42
UNIT 8: Splicing modulation therapy (fixing RNA) ....................................................... 46
UNIT 9: Genome editing therapy (fixing DNA) ............................................................. 52
BLOC IV: THERAPY FOR RENAL TUBULOPATHIES .......................................................... 57
UNIT 10: Renal physiology ........................................................................................ 57
UNIT 11: Current studies and therapy of renal tubulopathies ...................................... 62
UNIT 12: Drug-induced renal pathologies .................................................................. 68
BLOC V: MODERN CANCER THERAPIES ....................................................................... 71
UNIT 13: Basic knowledge RTK and cytokine signalling ............................................... 71
UNIT 14: Targeted cancer therapies ........................................................................... 81
BLOC VI: MODERN PAIN MANAGEMENT ....................................................................... 88
UNIT 15: GPCR-mediated signalling .......................................................................... 88
UNIT 16: Pain management (focus on GPCRs) ........................................................... 97
UNIT 17: Clinical aspects of pain management ........................................................ 104
2
, Molecular Therapy
BLOC I: DRUG DELIVERY AND DEVELOPMENT
UNIT 1: Drug delivery
1. General concepts in drug delivery
Drug delivery aims at achieving a high concentration of a drug molecule at the site of action
(absorption of the drug across a biological membrane). In this way, side effects, as for
example for toxic chemotherapeutics, can be reduced and the activity increased. Drug
delivery frequently is combined with targeting approaches.
- Characteristics of classical drugs:
o Small organic molecules.
o Lipophilic: to cross membranes (stomach, intestine...).
o Distribute freely through the body.
o Act by inhibiting enzymes/ receptors.
- Limitations of classical drugs:
o Chemical diversity limited: due to size and lipophilicity.
o Risk of side effects:
▪ Insufficient specificity: cross reactivity with other targets that are
more than one part of the body.
▪ Inhibition of important metabolic routes in non-target organs
(cancer).
o Poor inhibitors of protein-protein interactions are flat surfaces, so it is not as
easy to attach like deep pockets for binding.
o Oligonucleotides offer a very straight-forward way to inhibit or change
protein expression in a highly specific manner.
2. Drug targeting
Enhance the concentration of a drug at its site of action (target).
- Avoid side effects.
- Use expensive drugs more effectively:
o Less total dose.
o Higher concentration for longer period: protect from degradation and
excretion.
- For an established drug molecule.
Dual challenge
- Remain undetected until the target.
- Find and and hit your target.
3. The main components of a nanomedicine
- Surface modification: prevents rapid clearance.
- Targeting ligand: antibody, HER2 (breast cancer cells), growth factors (EGF-A), …
- Drug.
- Matrix: encapsulates and releases the drug. The ratio drug/matrix should be as hig
as possible.
o Liposome.
3
The benefits of buying summaries with Stuvia:
Guaranteed quality through customer reviews
Stuvia customers have reviewed more than 700,000 summaries. This how you know that you are buying the best documents.
Quick and easy check-out
You can quickly pay through credit card or Stuvia-credit for the summaries. There is no membership needed.
Focus on what matters
Your fellow students write the study notes themselves, which is why the documents are always reliable and up-to-date. This ensures you quickly get to the core!
Frequently asked questions
What do I get when I buy this document?
You get a PDF, available immediately after your purchase. The purchased document is accessible anytime, anywhere and indefinitely through your profile.
Satisfaction guarantee: how does it work?
Our satisfaction guarantee ensures that you always find a study document that suits you well. You fill out a form, and our customer service team takes care of the rest.
Who am I buying these notes from?
Stuvia is a marketplace, so you are not buying this document from us, but from seller mireiasabater24. Stuvia facilitates payment to the seller.
Will I be stuck with a subscription?
No, you only buy these notes for $6.86. You're not tied to anything after your purchase.
Can Stuvia be trusted?
4.6 stars on Google & Trustpilot (+1000 reviews)
56326 documents were sold in the last 30 days
Founded in 2010, the go-to place to buy study notes for 14 years now