Category Archives: AAV vectors

Gene editing target disease ?

Genome Editing with CRISPR-Cas9

Here’s a short list of some common diseases that might be curable or preventable with gene editing:

Breast cancer
Colon cancer
Huntington’s disease
Marfan, Parkinson’s
Prostate cancer
Retinitis pigmentosa
Sickle cell
Skin cancer
Duchenne muscular dystrophy
Color blindness
Cystic fibrosis
Down syndrome
Polycystic kidney
Turner syndrome.

There are hundreds of other more rare genetic disorders.… Read more

Gene therapy for spinal muscular atrophy (SMA) Type 1


Gene therapy is a well-suited approach for the treatment of SMA due to the monogenic nature of the disease—meaning it’s caused by the deletion of or mutations in a single gene. AVXS-101 is our clinical-stage, proprietary gene therapy candidate of a one-time, intravenous treatment for SMA Type 1—designed to prevent further muscle degeneration caused by SMA through:

Delivery of a fully functional human SMN gene into target motor neuron cells
Production of sufficient levels of SMN protein required to improve motor neuron function
Rapid onset of effect in addition to … Read more

Gene Therapy and regenerative medicine companies.



Tim Miller, Ph.D., President & CEO
Dallas, TX
PlasmaTech Biopharmaceuticals/Abeona Therapeutics is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech’s lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary

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