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

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

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

Development of New Gene Therapy Treatments

Exemplar Genetics ExeGen® Cystic Fibrosis Research Model Utilized in Development of New Gene Therapy Treatments

http://www.prnewswire.com/news-releases/exemplar-genetics-exegen-cystic-fibrosis-research-model-utilized-in-development-of-new-gene-therapy-treatments-300326909.html

Exemplar Genetics, a wholly owned subsidiary of Intrexon Corporation (NYSE:  XON) committed to enabling the study of life-threatening human diseases, today announced two publications in the current issue of the Journal of Clinical Continue reading Development of New Gene Therapy Treatments

UI strikes agreement with Pfizer Inc. to develop potential cystic fibrosis gene therapy

The University of Iowa Research Foundation recently finalized a license and sponsored research agreement with Pfizer Inc. to support the development of potential gene therapies for cystic fibrosis (CF) by the laboratories of the Continue reading UI strikes agreement with Pfizer Inc. to develop potential cystic fibrosis gene therapy

Gene therapy: A promising candidate for cystic fibrosis treatment

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Continue reading Gene therapy: A promising candidate for cystic fibrosis treatment

Spraying new genes into your lungs could ease misery of cystic fibrosis

For Oli Dillon, an effective therapy for cystic fibrosis can’t come soon enough. Thanks to a groundbreaking development, he’s already had a tantalising ‘taster’ of a treatment that could transform lives such as his. Continue reading Spraying new genes into your lungs could ease misery of cystic fibrosis