CRISPR and Gene Therapy

These flashcards cover the key concepts of CRISPR technology and gene therapy, their mechanisms, applications, challenges, and ethical considerations.

CRISPR

A genome editing technology that allows for precise alterations in DNA.

Genome Editing

The ability to make specific and intentional changes to the DNA of an organism.

Gene Therapy

A technique that uses genes to treat or prevent disease.

Selective Breeding

The process of choosing specific organisms to breed based on desired traits.

Artificial Selection

The intentional reproduction of individuals with desired traits.

DNA Endonucleases

Enzymes that cut DNA at specific sites.

CRISPR-Cas9

A system used for genome editing that consists of a guide RNA and the Cas9 endonuclease.

Gene Disruption

The alteration of a gene to render it nonfunctional.

Double-Strand Break Repair

A mechanism to fix breaks in both strands of DNA.

Homology Directed Repair (HDR)

A precise DNA repair process that uses a template to repair a double-strand break.

Non-Homologous End Joining (NHEJ)

A repair process that joins broken ends of DNA but may result in errors.

Intron Homing

A natural process where specific introns propagate within a population using endonucleases.

Homing Endonuclease

An enzyme that recognizes and cuts specific DNA sequences to promote intron spreading.

Zinc Finger Nucleases

Engineered proteins that create double-strand breaks at specific DNA sequences.

TALEN Nucleases

Engineered proteins that can be programmed to target specific DNA sequences for editing.

Base Editing

A technique that allows for the conversion of one DNA base pair into another without double-strand breaks.

dCas9

A 'dead' Cas9 protein that cannot cut DNA but can be used for regulation.

PAM Sequence

A short sequence adjacent to the target DNA that is required for Cas9 binding and cutting.

CRISPRoff

A technique for epigenetic modification that allows for reversible gene silencing.

CRISPRon

A method to activate gene expression using CRISPR technology.

Gene Augmentation

Adding a functional gene to compensate for a defective or missing gene.

Gene Replacement

Replacing a faulty gene with a functional one.

Targeted Cell Death

Inducing the elimination of specific cells, often used in cancer therapy.

Somatic Cells

Body cells that do not contribute to the next generation.

Germline Cells

Cells that produce gametes and can be passed to future generations.

In Vivo

Gene therapy conducted within a living organism.

Ex Vivo

Gene therapy done outside of the organism in controlled conditions.

Vectors

Carriers that deliver therapeutic genes into cells.

Adenovirus

A type of virus often used as a vector in gene therapy.

Adeno-Associated Virus (AAV)

A small virus used to deliver genetic material into cells.

Retrovirus

A virus that uses RNA as its genetic material and integrates into the host's genome.

HIV Vector

An engineered version of the HIV virus used to deliver therapeutic genes.

SCID

Severe Combined Immunodeficiency, a genetic disorder treated with gene therapy.

ADA SCID

A form of SCID caused by a mutation in the ADA gene.

Ethical Concerns

Moral implications surrounding gene editing, including safety and societal impacts.

Base Excision Repair

A DNA repair process that removes and replaces damaged bases.

CRISPR Cas12a

A variant of Cas9 that functions similarly in genome editing.

Off-Target Effects

Unintended edits in the genome that occur due to imprecise cutting.

Methylation

The addition of a methyl group to DNA that can inhibit gene expression.

Epigenome Editing

Modifying the epigenetic markers that regulate gene expression.

Nuclease-Free Editing

Gene editing techniques that do not rely on cutting DNA.

Hybrid Protein

A protein that combines functional domains from different sources.

CRISPR Systems

Different configurations and components of the CRISPR technology used for genome editing.

Transactivating RNA (tracrRNA)

An RNA that is essential for processing the pre-crRNA in the CRISPR system.

Gene Regulation

Controlling the expression of genes to enhance or suppress their activity.

Immune Response

The body's defense mechanism against pathogens or foreign entities.

CRISPR Clinical Trials

Research studies that investigate the safety and effectiveness of CRISPR technologies in treating diseases.

CRISPR Technology Challenges

Issues such as delivery efficiency, off-target effects, and ethical considerations.

Genome-wide Screening

A method to analyze many genes simultaneously to identify effects or interactions.

RNA-guided Endonuclease

An enzyme that uses an RNA molecule to locate its target in the genome.

Precision Medicine

A medical model that proposes the customization of healthcare, with medical decisions tailored to individual patients.

Phage Therapy

The use of bacteriophages to treat bacterial infections.

Introns

Non-coding sequences of DNA that are removed during mRNA processing.

Exons

Coding sequences of DNA that remain in the final mRNA.

Spliceosome

A complex that removes introns from pre-mRNA.

Gene Editing Efficacy

The effectiveness and precision with which gene editing tools can alter genomes.

CRISPR Genome Editing Timeline

The historical development and advancements in CRISPR technologies.

Targeted Gene Therapy

A gene therapy approach that focuses on specific genes related to diseases.

Fetal Hemoglobin

A type of hemoglobin that can be induced to counteract sickle cell disease.

CRISPR Therapeutics

Companies focused on translating CRISPR technologies into clinical applications.

Hybrid Vectors

Vectors combining elements of viral and non-viral methods for gene delivery.

Patient-Specific Treatments

Custom therapies designed based on the genetic profile of individual patients.

Bioethics

The study of ethical issues emerging from advances in biology and medicine.

Genome Editing Techniques

Various methods, including CRISPR and TALEN, used for gene editing.

Neural Stem Cells

Stem cells that can differentiate into neurons and other cell types in the nervous system.

Transcription Factors

Proteins that regulate gene expression by binding to specific DNA sequences.

Gene Repair Mechanisms

Natural processes cells use to repair damaged DNA.

CRISPR Applications

The various uses of CRISPR technology in research, medicine, and agriculture.

Genomic Library

A collection of the total genomic DNA from a single organism.

Homology-Based Editing

Techniques relying on homologous sequences to make precise genetic modifications.

Cis-Elements

Regulatory regions of DNA that influence the expression of associated genes.

Trans-Elements

Molecules, usually proteins, that interact with cis-elements to regulate gene expression.

Cancer Gene Therapy

Gene therapy applied to treat or prevent cancer.

Regulatory Approval

The process by which health authorities evaluate and approve new therapies.

Genetic Compensation

A phenomenon where other genes compensate for the loss of function in a mutated gene.

Delivery Mechanisms

Methods and strategies for transporting therapeutic genes into target cells.

Plasmids

Small circular DNA molecules used as vectors to introduce foreign DNA into cells.

Antisense Oligonucleotides

Short DNA or RNA molecules designed to bind to specific mRNA.

Immunotherapy

A treatment that uses the body's immune system to fight diseases, particularly cancer.

Gene Silencing

Techniques to decrease or prevent the expression of a gene.

Genetic Modification

An alteration of the genetic material of an organism.

Clinical Research

Studies conducted to evaluate new medical treatments and therapies.

Proof of Concept

Demonstration that a method operates as intended, often used in pilot studies.

Transgenic Organisms

Organisms that contain a gene or genes which have been artificially inserted.

Molecular Cloning

The process of making multiple copies of a specific DNA fragment.

Gene Expression Profiling

A method to study the expression of multiple genes at once.

Therapeutic Genes

Genes that are introduced into cells to treat or prevent diseases.