분자유전학실험실 (단국대학교 분자생물학과)



 이성욱 ( 2017-10-14 01:20:21 , Hit : 665
 Advisors to FDA Recommend Approval of Gene Therapy for Blindness

The Scientist »
The Nutshell


http://www.the-scientist.com/?articles.view/articleNo/50623/title/Advisors-to-FDA-Recommend-Approval-of-Gene-Therapy-for-Blindness/&utm_campaign=NEWSLETTER_TS_The-Scientist-Daily_2016&utm_source=hs_email&utm_medium=email&utm_content=57334565&_hsenc=p2ANqtz-_UKkXVdsS3kZ4dscjaPo-FJpNONAY87gv0qmYNW2FwQqK-IElHOva7yGr6-TprrQvboc_oH4Fo81WWeKZPgFs1fXzDaw&_hsmi=57334565



Spark Therapeutics’s Luxturna would be the first approved therapy in the U.S. that replaces or repairs a defective gene inherited from one’s parents.

By Jef Akst | October 12, 2017



AddThis Sharing Buttons
Share to FacebookShare to TwitterShare to RedditShare to 이메일Share to 더 보기



gene therapy for blindness
FLICKR, HELGA BIRNA JÓNASDÓTTIR
A panel of 16 independent advisers to the US Food and Drug Administration (FDA) voted unanimously today (October 12) to recommend the approval of Luxturna, a gene therapy meant to treat Leber congenital amaurosis—a rare, inherited form of childhood blindness—and other retinal disorders, STAT News reports. If the agency agrees with the panel’s recommendation, Luxturna would be the first gene therapy aimed at correcting a congenital defect approved in the U.S.

The therapy, developed by Philadelphia-based Spark Therapeutics, is injected directly into the eye in a one-time treatment. Billions of virus particles deliver a functional copy of the RPE65 gene—which encodes a protein necessary for normal vision and is mutated in patients with certain vision disorders—to the retina. So far, more than two dozen children and adults with RPE65 mutations have been treated with Luxturna in the context of clinical trials, and some 93 percent of them have shown improved light sensitivity and functional vision. The effects are long lasting, with improvements continuing for at least four years in some patients that have been tracked that long.

“If my child or myself had this condition I would not hesitate for a moment getting treatment with [Luxturna],” Albert Maguire, a retinal disease specialist at Children’s Hospital of Philadelphia who led the Luxturna clinical trials, said at the advisory panel hearing today, according to STAT News.

If approved, Luxturna would be the first gene therapy that fixes an inherited genetic disorder in the U.S. (The official title of first approved gene therapy goes to Novartis’s CAR T-cell therapy, greenlighted this August.) The FDA must make its decision by January 12, 2018.

“This is what I believe medicine is going to be like for the next 20, 30, if not 50 years,” Spark CEO Jeff Marrazzo tells MIT Technology Review of these types of gene therapies. “I think this is the beginning of an age that is going to fundamentally change medicine.”







1227   DNA Repair Mechanism Aids Cancer by Mending Chemo Damage  이성욱 2017/11/10 569
1226   Alcoholism Gene Therapy Shows Promise  이성욱 2017/09/20 582
1225   CRISPR Used in Human Embryos to Probe Gene Function  이성욱 2017/09/22 583
1224   암 면역요법, 일부 환자에게만 효과 있는 이유  이성욱 2018/01/12 589
1223   [바이오토픽] CRISPR 기반 유전자치료를 방해하는 면역계  이성욱 2018/01/10 591
1222   Nature Methods retracts CRISPR study  이성욱 2018/04/10 600
1221   From Startups to Moguls] CRISPR 유전자가위 없이 편집한다? Homology Medicines  이성욱 2017/09/11 605
1220   FDA Approves Gilead CAR-T Therapy Yescarta, Plans Regenerative Medicine Policy 'Soon'  이성욱 2017/10/20 610
1219   Severe Toxicity Reported in High-Dose AAV Gene Therapy in Animals  이성욱 2018/02/01 614
1218   CRISPR System Targets RNA in Mammalian Cells  이성욱 2017/10/06 640
1217   DNA Construction  이성욱 2017/09/15 660
  Advisors to FDA Recommend Approval of Gene Therapy for Blindness  이성욱 2017/10/14 665
1215   2018 Lasker Award Winners: Gene Expression Researchers, Propofol Discoverer, Pioneer for RNA Biology, and Women in STEM  이성욱 2018/09/12 671
1214   [바이오토픽] 체크포인트 저해제의 부작용: 자가면역질환 초래  이성욱 2017/11/16 689
1213   First CAR T-Cell Therapy Approved in U.S.  이성욱 2017/09/01 691
1212   UPDATED: FDA ushers in a new era in cancer treatment with ‘historic’ CAR-T approval for Novartis  이성욱 2017/08/31 696
1211   Tunable Form of CRISPR Is an Efficient Regulator of Stem Cell Expression  이성욱 2016/03/14 701
1210   [바이오 업계 소식: From Startups to Moguls] CRISPR-Cas9에 대한 경고와 업계 반응  이성욱 2017/06/14 701
1209   Mammalian Immunity: What’s RNAi Got to Do with It?  이성욱 2017/07/25 704
1208   코오롱 인보사허가  이성욱 2017/07/12 706

[1][2] 3 [4][5][6][7][8][9][10]..[64] [다음 10개]
 

Copyright 1999-2021 Zeroboard / skin by ROBIN