Lecture

Gene Therapy: Targets and Therapeutics

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In course

BIO-478: Pharmacology and pharmacokinetics

This course introduces the student to the fudamentals of pharmacology, pharmacokinetics and drug-receptor interactions. It discusses also pharmacogenetics and chronopharmacology, to exemplify the challenges of personalized medicine. ...

Description

This lecture covers gene therapy targets, including receptors, enzymes, ion channels, and protein therapeutics. It discusses gene therapy principles, delivery challenges, and successes in treating monogenic diseases like spinal muscular atrophy. The use of RNA-based therapeutics, such as antisense inhibitors and RNA interference, is also explored, with examples like Nusinersen for SMA and Patisiran for hereditary transthyretin-mediated amyloidosis. The lecture delves into the history of gene therapy, from the first cure in a mouse to recent FDA-approved treatments like Luxturna and Kymriah. It highlights the challenges and advancements in gene therapy, including the development of CAR T cell therapy and CRISPR-based treatments for genetic disorders like Leber congenital amaurosis.

Instructors (2)

Romain Zufferey

Teaching & PhD Courses Biology for engineers MSE-212 This course consists of an introduction to biology and more particularly to biology as a multidisciplinary field, emphasizing natural examples of materials engineering. It should therefore allow engineering students to find inspiration from biology in future materials research problems. Cellular biology and biochemistry for engineers BIO-105 Basic course in biochemistry as well as cellular and molecular biology for non-life

Dmitri Firsov

Teaching & PhD Courses Pharmacology and pharmacokinetics BIO-478 This course introduces the student to the fudamentals of pharmacology, pharmacokinetics and drug-receptor interactions. It discusses also pharmacogenetics and chronopharmacology, to exemplify the challenges of personalized medicine.

Official source

Related concepts (31)

Genetic disorder

A genetic disorder is a health problem caused by one or more abnormalities in the genome. It can be caused by a mutation in a single gene (monogenic) or multiple genes (polygenic) or by a chromosomal abnormality. Although polygenic disorders are the most common, the term is mostly used when discussing disorders with a single genetic cause, either in a gene or chromosome. The mutation responsible can occur spontaneously before embryonic development (a de novo mutation), or it can be inherited from two parents who are carriers of a faulty gene (autosomal recessive inheritance) or from a parent with the disorder (autosomal dominant inheritance).

Genetic testing

Genetic testing, also known as DNA testing, is used to identify changes in DNA sequence or chromosome structure. Genetic testing can also include measuring the results of genetic changes, such as RNA analysis as an output of gene expression, or through biochemical analysis to measure specific protein output. In a medical setting, genetic testing can be used to diagnose or rule out suspected genetic disorders, predict risks for specific conditions, or gain information that can be used to customize medical treatments based on an individual's genetic makeup.

Genetic counseling

Genetic counseling is the process of investigating individuals and families affected by or at risk of genetic disorders to help them understand and adapt to the medical, psychological and familial implications of genetic contributions to disease. This field is considered necessary for the implementation of genomic medicine.

CRISPR

CRISPR (ˈkrɪspər) (an acronym for clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of bacteriophages that had previously infected the prokaryote. They are used to detect and destroy DNA from similar bacteriophages during subsequent infections. Hence these sequences play a key role in the antiviral (i.e. anti-phage) defense system of prokaryotes and provide a form of acquired immunity.

Spinal muscular atrophy

Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic cause of infant death. It may also appear later in life and then have a milder course of the disease. The common feature is progressive weakness of voluntary muscles, with arm, leg and respiratory muscles being affected first.

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