PBS’s Documentary ‘The Gene Doctors’ Arrives Amid A Gene Therapy Boom

Molly Troxel, who has an inherited degenerative eye disease, regained some vision after being treated with Spark’s experimental gene therapy, Luxturna. (Photo courtesy of PBS.)

This month, PBS is airing a documentary called The Gene Doctors that spotlights several emerging gene therapies, Continue reading PBS’s Documentary ‘The Gene Doctors’ Arrives Amid A Gene Therapy Boom

Cystic Fibrosis Foundation to Give $3 Million for Novel CFTR Therapeutic

Arcturus Therapeutics, Inc. (“Arcturus” or the “Company”), a leading RNA medicines company, today announced that it has entered into a research agreement with Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation in which CFFT will pay up to $3 million to advance LUNAR-CF, a novel messenger RNA (mRNA) therapeutic formulated with Arcturus’ LUNAR™ delivery technology.

Continue reading Cystic Fibrosis Foundation to Give $3 Million for Novel CFTR Therapeutic

Scientists turn human induced pluripotent stem cells into lung cells: ‘Bronchospheres’ may pave way for personalized cystic fibrosis treatments — ScienceDaily

https://www.sciencedaily.com/releases/2017/05/170503131922.htm

For years, scientists who study lung diseases like cystic fibrosis have tried to track this process in detail, from start to finish, in the hope that understanding how lungs form normally may help explain how things go wrong. Now, scientists at Boston University’s Center for Regenerative Medicine (CReM) have announced Continue reading Scientists turn human induced pluripotent stem cells into lung cells: ‘Bronchospheres’ may pave way for personalized cystic fibrosis treatments — ScienceDaily

You are invited to attend a free web-cast event SATURDAY, APRIL 22, 2017

RARE LUNG DISEASES PATIENT EDUCATION DAY ON CYSTIC FIBROSIS, CHILDREN’S INTERSTITIAL LUNG DISEASE AND PRIMARY CILIARY DYSKINESIA

REGISTER NOW AT:
https://attendee.gotowebinar.com/register/6993341835513746691

This event is co-sponsored by the American Thoracic Society, the Cystic Fibrosis Foundation, the Children’s Interstitial Lung Disease Foundation and the Primary Continue reading You are invited to attend a free web-cast event SATURDAY, APRIL 22, 2017

Cystic fibrosis and sickle-cell anemia could be corrected in embryos

https://www.geneticliteracyproject.org/2017/03/08/cystic-fibrosis-sickle-cell-anemia-could-be-corrected-in-embryos-with-new-crispr-variant/

Cystic fibrosis, sickle-cell anemia could be corrected in embryos with new CRISPR variant

Since the discovery of the genome-editing tool CRISPR/Cas9, scientists have been looking to utilize the technology to make a significant impact on correcting genetic diseases. Technical challenges have made it Continue reading Cystic fibrosis and sickle-cell anemia could be corrected in embryos

Scientists use ‘molecular-Lego’ to take CRISPR gene-editing tool to the next level

http://www.medicalnewstoday.com/releases/314716.php

A team of researchers at Western University is playing with molecular-Lego by adding an engineered enzyme to the revolutionary new gene-editing tool, CRISPR/Cas9. Their study, published in the Proceedings of the National Continue reading Scientists use ‘molecular-Lego’ to take CRISPR gene-editing tool to the next level

My Five Takeaways from #NACFC2016

I had a dream last night, and in that dream the Limp Bizkit classic song, Nookie was on repeat for some reason. I have no idea why; I haven’t heard that song in about 12 years. Needless to say I woke up pretty jacked up and motivated to get sh*t done before the Packers game this afternoon. Continue reading My Five Takeaways from #NACFC2016

Gene Therapy for Treating CF

http://www.news-medical.net/news/20160920/Gene-therapy-may-be-viable-approach-for-treating-CF-lung-problems.aspx

Gene therapy may be viable approach for treating CF lung problems

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis Continue reading Gene Therapy for Treating CF

Gene therapy for treating CF lung problems

Gene therapy may be viable approach for treating CF lung problems

http://www.news-medical.net/news/20160920/Gene-therapy-may-be-viable-approach-for-treating-CF-lung-problems.aspx

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF).

Working with CF pigs, the researchers, based in the UI Pappajohn Biomedical Continue reading Gene therapy for treating CF lung problems

Announcing New Clinical Trial Tools From The CFF

Today, we are thrilled to announce that we have launched three new clinical trial tools to better inform our community about the significance of clinical research and help people with CF find the trials that are right for them. We hope that these Continue reading Announcing New Clinical Trial Tools From The CFF