Gene Editing and Gene Therapy Breakthroughs Propelling Mainstream Treatments

Tuesday, October 16, 2018 Genetics & Stem Cells News
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NEW YORK, Oct. 15, 2018 /PRNewswire/ -- A new wave of gene therapy treatments to revolutionize treatment of inherited

diseasesRead the full report: https://www.reportlinker.com/p05589919 The drug approvals of Yescarta (axicabtagene ciloleucel), Luxturna (Voretigene neparvovec) and Kymriah (Tisagenlecleucel) have turned a leaf in the pharmaceutical chapter
of gene therapy development.Currently, genetic disorders do not have satisfactory treatment outcomes.This is in part due to the inability of small molecule drugs and enzyme replacement therapies, usually prescribed for inherited diseases, to correct the root cause of the disorders.In other words, they are not corrective therapies.Gene therapy offers exactly what small molecule drugs and enzyme therapy therapies don't- they can offer corrective solutions for inherited disorders.A new wave of gene therapy breakthroughs can be expected to follow in the next few years after a two-decade long hiatus that witnessed very few proven gene therapy drug candidates.This potential in-flux of gene therapy candidates is due to the two fundamental factors.First, the refining and development of cutting-edge vector platform both by academia and by pharmaceutical industry.This has resulted in the widely used advanced Adeno-associated viral vectors and Lentiviral vectors, both of which are used according to the applicability.Second, the development of novel gene editing tools such as TALENs, Meganucleases, CRISPR/Cas along with the improvement of old techniques such as ZFNs have helped improve the translational capability of gene therapies currently in clinical development.In the following study, the development of gene editing tools as well as gene-transfer enabling vectors is traced over the last decade or so while also putting into perspective the number of gene therapy candidates currently in clinical development in addition to the various potential cures and satisfactory treatments that await patients affected by genetic diseases .Read the full report: https://www.reportlinker.com/p05589919 About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place. __________________________ Contact Clare: clare@reportlinker.com US: (339)-368-6001 Intl: +1 339-368-6001

Cision View original content:http://www.prnewswire.com/news-releases/gene-editing-and-gene-therapy-breakthroughs-propelling-mainstream-treatments-300731023.html

SOURCE Reportlinker



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