Cell and Gene Therapy for Spine Regeneration
This article provides an evidence-based personal perspective on the future of cell and gene therapy for degenerative diseases of the intervertebral disc. This paper focuses on how mammalian protein production platforms and transfected and irradiated protein packaging cell lines may be used as “cellular factories” for overproduction of therapeutic proteins and proanabolic growth factors, particularly in the context of regenerative therapies. This paper also speculates on future opportunities and challenges in this area of research and how new innovations in biotechnology affect cell a nd gene therapy for degenerative diseases.
Abeona Therapeutics announced that the U.S. FDA has removed its clinical hold on the company ’s pivotal Phase 3 clinical trial evaluating gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
For the 10th time this year, Roche has extended its $114.50 per share tender offer to Spark Therapeutics stockholders. In February, Roche of Basel, Switzerland, entered into an agreement to buy Philadelphia gene therapy pioneer Spark Therapeutics for $4.3 billion. The latest offer by Roche was set to expire Dec. 10, but has now been extended to 5 p.m. on Dec. 16. Roche and Spark once again said the offer was ex tended to provide additional time for the Federal Trade Commission and the UK Competition…
Latest treatments with price tags as high as $2m require new financing models
Single treatment using advanced microsurgery can cure condition for life
Rapid advances in technology from bioelectronics to gene therapy offer new ways to treat many illnesses. But medical breakthroughs bring with them new questions of pricing, access and scientific responsibility
Discussion Gaucher disease (GD) was first described by Philippe Gaucher in 1882. It was the first lysosomal storage disease (LSD) described and is the comparison prototype for many variations and their treatment. There are about 50 LSD and more well-known ones include Fabry, Niemann-Pick and Pompe diseases. LSDs currently have more than 300 different enzymes or membrane proteins affected which cause central nervous system and visceral disease. Overall the frequency of LSDs in aggregate is 1:3000 – 7000 live births. GD has an estimated prevalence of 1:57,000 – 111,000. It is higher within the Ashkenazi Jewish po...
Conclusion:In this study, we demonstrated that the combination of neuron-specific enolase promoter and valproic acid induced gene overexpression in induced neural stem cells under hypoxic conditions and also in spinal cord injury depending on valproic acid administrationin vivo. Combination of valproic acid and neuron-specific enolase promoter in induced neural stem cells could be an effective gene therapy system for hypoxic spinal cord injury.
Gene Therapy, Published online: 09 December 2019; doi:10.1038/s41434-019-0116-1The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model
Gene Therapy, Published online: 09 December 2019; doi:10.1038/s41434-019-0107-2An engineered serum albumin-binding AAV9 capsid achieves improved liver transduction after intravenous delivery in mice