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Learn More - Healing Genes
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Gene Therapy

Medline Plus

MedlinePlus links to health information from the National Institutes of Health, other federal agencies, and non-governmental web sites. You’ll learn more about genes and gene therapy here.

Learn Genetics​

The University of Utah explains gene therapy, methods for delivering genes into cells, and helps teachers explain genetics. You can even play Space Doctor with your favorite aliens!

 

Genetics Home Reference What is Gene Therapy

The Genetics Home Reference explains gene therapy – how it works, is it safe, is there a gene therapy available for a specific disease or condition.

Centre for Genetics Education

This fact sheet describes gene therapy as it is used to treat medical conditions and the benefits and challenges it raises. It provides a brief overview that is quite helpful.

 

 

Your Genome

If you want to learn gene therapy in layman’s terms, including gene augmentation therapy, inhibition therapy, and DNA transfer, this site is worth exploring. We love the bullet point explanations.

KidsHealth®​

Designed for families this information provides a gene therapy primer for parents who may be considering gene therapy as an option for their child.

 

What is Gene Therapy?​

OncoLink outlines the role gene therapy plays in the fight against cancer.  Here, you’ll receive a brief but amazingly thorough background on what’s happening to genes during cancer, gene therapy, delivery, and side effects.

Cancer Gene Therapy and Cell Therapy​

To learn the differences among gene therapy, cell therapy and gene editing, be sure to visit the Different Approaches section in Gene Therapy 101.  You’ll also separate myths from facts in the Gene Therapy Basics section.

Gene Editing

CrispR Gene Editing

Gene Therapy Inside and Out

Source: FDA

CrispR Decoded

Source: techologyreview.com

Cell Therapy

Stem Cell Basics​

National Institutes of Health (NIH) Stem Cell Information introduces you to stem cells and their unique properties. In addition, you can stay more current on NIH research.

Stem Cell Facts​

Learn about stem cells, their potential and limitations as treatment options, and how today’s research leads to tomorrow’s medicine.

The Power of Stem Cells

An overview of stem cell use in medicine, including helpful video links, this site introduces stem cell basics, addresses what happens when your immune system rejects cell-based therapies, and helps you evaluate the benefits and risks of stem cell therapy.

Types of Stem Cells​

Learn the basic differences among self-renewing cells, including embryonic, tissue-specific, mesenchymal, and induced pluripotent stem cells.

What Diseases and Conditions can be Treated with Stem Cells?​

What do we know? What are researchers investigating? What are the challenges in stem cell medicine? You’ll find more information on these important topics here.

Stem cells: What they are and what they do​

Learn about stem cell types, current and possible uses, ethical issues, and the state of research and practice.

Stem Cells Explained

Source: Elements Science

Stem Cells

Source: Khan Academy Medicine

Defeating Sickle Cell Disease

Source: CIRM

Stem Cells: An Introduction

Source: Elements Science

Induced Pluripotent Stem Cells

Source: Khan Academy Medicine

Vectors

Viral Vectors and Their Promise…​

A brief history plus application of viral vectors in gene therapy – if you’re comfortable with science.

Viral Vectors in Gene Therapy​

Although a scientific article, you benefit from a good list of vector features, which helps you understand why a researcher may select a specific viral vector for particular gene therapy.

Introduction to Vectors​

If you seek a broader introductory view of vectors, you can learn about both viral and non-viral vectors used in gene therapy here.

Viral Vectors

Source: Aoife Scanlon

Example of Gene Therapy with Viral Vectors

Source: AGTC

How AAV Gene Transfer Works

Source: UniQure

All gene therapy products need help to get to the right location in the body. A gene that is inserted directly into a cell usually does not function. Instead, a transport vehicle called a vector is genetically engineered to deliver the gene,

Some viruses are used as vectors because viruses can deliver a new gene by infecting the cell. Imagine: Researchers are turning the virus into the helper!

 

VIRAL VECTORS

Come again – why do doctors want to use a virus to transfer the good gene from the laboratory to the patient?

When a virus attacks a person, it inserts genetic material into a targeted cell. This genetic material includes basic instructions that trick the body into producing more copies of the virus. Some viruses (called retroviruses and lentiviruses) can even integrate their genes into a patient’s own strands of genetic code.

Medical researchers realized that if they could get rid of the disease-causing part of the virus yet keep the equivalent of its delivery truck, motor and GPS (navigation) system, they could use the virus to transport healthy genes into the body instead, and the body wouldn’t be so eager to reject the new healthy genes – maybe.

Scientists are very careful to select the virus shuttle they think will perform the best given the patients’ needs.

Three examples:

  • Some viruses don’t integrate into the body’s own strands of genetic code, and, thus, don’t linger inside the body.
  • Some viruses are slower to act, can integrate into the body’s genetic code, and tend to stimulate the body’s natural immune response, which may be helpful for patients who suffer from debilitating lifetime issues, such as Hemophilia.
  • Some viruses can carry a much larger genetic payload than others.

What are the risks?

There are risks associated with using viral vectors to transport genes into the body.

  • Rarely, a new gene may end up in the wrong DNA location, which could cause an unintended mutation, even cancer.
  • The introduced genes could cause an unwanted, aggressive immune reaction.
  • The science is new enough that there could be unintended and unforeseen consequences that we haven’t identified yet.

How are scientists protecting us?

Gene therapy must pass through the same clinical trial steps as prescription drugs. It must be thoroughly tested for patient safety.

Technology

Genome Editing: Technology Primer​

If you’re looking for easy-to-understand descriptions, this is a great place to start to learn more about the technologies used in genome editing.

ZFNS, TALENS and CRISPR

Learn more about the technologies that have revolutionized genomic engineering, allowing professionals to discover potential mechanisms that may stop DNA-based diseases.

Genome Editing: Which Should I Choose…​

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CRISPR Gene Editing

Source: Nature Video

How CRISPR lets us edit our DNA

Source: TED

Method of the Year 2011: Gene-Editing Nucleases

Source: Nature Video