Human Gene Therapy Flashcards

F Gene Therapy

A technique that modifies a person's genes to treat or cure diseases by replacing disease-causing genes with healthy copies or inactivating malfunctioning genes.

Gene Therapy

China

Which was the first country to approve a gene therapy-based product for clinical use?

Adenosine deaminase deficiency (ADA deficiency)

What was the first therapeutic gene transfer used to treat?

Adenovirus vector

What type of vector was used in the first officially approved gene transfer into humans?

First directly administered gene therapy, voretigene neparvovec, for retinal dystrophy

What type of therapy was approved in the US in December 2017?

63.3%

What percentage of gene therapy clinical trials are conducted in the USA?

America

Which continent accounts for the highest percentage of gene therapy clinical trials?

Cancer diseases

Which type of disease is most addressed by gene therapy clinical trials?

Direct Delivery and Cell-Based

What are the two main types of Gene Therapy Delivery?

Gene Replacement Therapy and Gene Addition

What are the two approaches to gene therapy?

Replacing a mutated gene with a healthy gene.

What does Gene Replacement Therapy involve?

Gene Inhibition or Knockdown and Gene Editing

What are additional approaches to Gene Therapy?

To provide sufficient gene expression in enough targeted cells to ameliorate or correct dysfunctional phenotype.

What is the aim of Gene Replacement Therapy (GRT)?

Recessive disorders, single gene mutations, and X-linked diseases

What types of diseases is Gene Replacement Therapy suitable for?

Retrovirus, Lentivirus, Herpes Simplex Virus type 1 (HSV-1), Adeno-Associated Virus (AAV), and Adenovirus.

What are the 5 main classes of Viral Vectors?