Education

Watch “Understanding the Gene Therapy Process and Aftercare,” from ASGCT and NORD

ASGCT Staff | December 06, 2019

The webinars in this series are designed to inform patients, patient advocates, families, and the public about various aspects of gene therapy.

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Learn About Blood Cancers and CAR T-Cell Therapy

ASGCT Staff | December 03, 2019

CAR T-cell therapy is a treatment approach that helps the immune system recognize cancer cells as a threat and eliminate them.

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Learn About Gene Therapy for Pompe Disease

ASGCT Staff | November 19, 2019

Pompe disease occurs when the genes that instruct our cells to create the GAA enzyme are faulty. The GAA enzyme’s job is to break down complex molecules like glycogen, which is a substance that helps our body store energy.

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Watch “The FDA's Role in Gene Therapy,” from ASGCT and NORD

ASGCT Staff | November 15, 2019

The webinars in this series are designed to inform patients, patient advocates, families, and the public about various aspects of gene therapy.

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Learn About Gene Therapy for Hemophilia

ASGCT Staff | November 12, 2019

Hemophilia occurs when the genes that are vital to producing clotting factors are faulty. There are existing treatments for hemophilia that require lifelong administration, while gene therapy may offer a one-time treatment to address the cause of the disease. 

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How Can Gene Therapy Help MPS Patients?

ASGCT Staff | October 29, 2019

There are seven different types of MPS, each caused by a different faulty gene. Gene therapy may be able to slow the progression of the MPS disorders I, II, III and IV, although it cannot reverse damage the disease has already caused. 

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Learn About Gene Therapy for Huntington’s Disease

ASGCT Staff | October 15, 2019

The American Society of Gene & Cell Therapy (ASGCT) released its latest resource profiling disease-specific treatment efforts, this time focusing on developing gene therapies for Huntington’s disease.

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ASGCT Education Program Returns, Covers Parkinson's Disease

ASGCT Staff | October 10, 2019

ASGCT is proud to launch a second round of disease-specific resources in its Patient Education program. The first of the seven new resources focuses on developing gene therapies for Parkinson’s disease.

ASGCT News   |   Education
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Watch “The Science Behind Gene Therapy,” from ASGCT and NORD

ASGCT Staff | October 08, 2019

The webinars in this series are designed to inform patients, patient advocates, families, and the public about various aspects of gene therapy.

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Rhoda Baer acquired from National Cancer Institute

Bridging the Gap Between Scientists And Patients In Gene Therapy Development

David Barrett, J.D. | September 27, 2019

Patients with rare diseases must rely collectively on scientists, physicians, pharmaceutical and biotechnology companies, and rare disease advocates to learn about these disorders, says ASGCT Executive Director David Barrett.

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FDA photo

Margaret Hamburg, M.D. to Speak at Policy Summit

ASGCT Staff | September 26, 2019

Margaret Hamburg, M.D., an internationally recognized expert in medicine and public health, will be one of the keynote speakers on November 6, the third and final day of the ASGCT Policy Summit in Washington, D.C.

 

Education   |   Policy Summit   |   Policy Updates
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Watch “Gene Therapy: Yesterday, Today, and Tomorrow” from ASGCT and NORD

ASGCT Staff | September 24, 2019

“Gene Therapy: Yesterday, Today, and Tomorrow,” the first of five educational webinars from ASGCT and the National Organization for Rare Disorders (NORD), is now available for free.

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Dose escalation of AAV-PHP.B in the mouse brain following intravenous and intra-arterial delivery

Effectiveness of Gene Therapy Delivery to the Brain Varies by Species

Edith Pfister, Ph.D. | September 19, 2019

In reviewing a recent Molecular Therapy article, Edith Pfister, Ph.D., associate editor of ASGCT's newsletter The Vector, outlines patterns of virus CNS transduction efficiency in non-human primates in comparison with mice.

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Credit: Daniel Soñé Photography, LLC

NIH Director Collins to Speak at ASGCT Policy Summit

ASGCT Staff | September 05, 2019

Francis Collins, M.D., Ph.D., director of the National Institutes of Health, will deliver his keynote address, "The Need for an International Moratorium on Clinical Uses of Human Germline Gene Editing," on day three of the ASGCT Policy Summit, November 6.

Education   |   Policy Summit   |   Policy Updates
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ASGCT, NORD Partner for Free Educational Gene Therapy Webinars

ASGCT Staff | August 19, 2019

Have you heard about gene therapy? Do you want to learn about the history of gene therapy, which gene therapies are currently available and what the future may hold? ASGCT and NORD partner to host the first of five educational gene therapy webinars, "Gene Therapy: Yesterday, Today and Tomorrow," on August 29 at noon EST.

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How Sonic Hedgehog Explains Age-Related Stem Cell Function and Fertility Issues

Edith Pfister, Ph.D. | July 23, 2019

In reviewing a recent Molecular Therapy article, Edith Pfister, Ph.D., associate editor of ASGCT's newsletter The Vector, outlines the possible effects of stem cell function on age-related fertility issues.

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2020

23rd Annual Meeting

May 12-15 | Boston