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In vivo Reprogramming

In vivo reprogramming is a therapeutic approach to regenerative medicine aimed at delivering a cell-based therapy to an organ or tissue in need of functional restoration without the use of a cellular agent. Building on principles of cellular reprogramming, epitomized by Yamanaka’s Nobel-winning discovery of ‘Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors’ in 2007, in vivo reprogramming employs transdifferentiation, or direct conversion of a source cell type into a target cell type without an intermediate pluripotent or stem cell stage, to affect the conversion of cells directly in the human body.

The rationale for in vivo reprogramming arises from the bottlenecks associated with the development of ex vivo cell therapies – manufacturability, scalability and the risk of immune rejection. Despite progression toward allogeneic development, the need to replicate the cell’s in vivo microenvironment in in vitro conditions and process variation between cell type, indication or disease area will remain a challenge.

In vivo reprogramming leverages the plasticity of cells and methods for the induction of cellular identity genes, such optimal combination of transcription factors, to achieve cellular reprogramming directly in situ, whilst harnessing the innate microenvironment to maintain functional maturity. The method has the potential to circumvent the need for ex vivo cell manufacture and the risk of immune rejection. Research into in vivo reprogramming methodologies includes the in situ generation or rejuvenation of cardiomyocytes, pancreatic β cells and neurons for regenerative medicine, as well as muscle regeneration and rejuvenation/anti-aging studies via partial reprogramming using the OKSM factors.

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In vivo Reprogramming

Here we discuss advances in in vivo reprogramming techniques, such as the MOGRIFY® platform, which enable the prediction of key regulatory factors or small molecules required to drive cell reprogramming of any mature cell type to any other cell type, sourcing the optimal cell conversions in situ.

White Paper
Realizing Retinal Regeneration: How can data-driven in vivo reprogramming be used to treat retinal and optic nerve degeneration?
2020   |   Evdokia Paza, Alice Lightowlers, Tim Landy, Geraint Parfitt
Approximately 1 in 2,000 people worldwide are affected by inherited retinopathies but few treatment options are available for retinal degeneration. The FDA’s 2017 approval of LUXTURNA to treat inherited retinal dege...
Application Note
Accelerating Regenerative Medicine Approaches to Type 1 Diabetes Through Direct Cell Reprogramming
2020   |   Lawrence Billing
Current treatment of type 1 diabetes mellitus (T1DM) depends on regular subcutaneous injections of exogenous insulin. Unfortunately, exogenous insulin therapy is associated with patient compliance issues and life-thre...
Webinar
Webinar: A Systematic Approach for Driving Cell Identity and Accelerating Regenerative Medicine
2020   |   Aida Moreno-Moral, Pierre-Louis Joffrin
Despite the 2007 discovery for converting a somatic cell into an induced pluripotent stem cell (iPSC) using transcription factors (TFs), the requirement to recapitulate biological pathways in the reprogramming of iPSC...
Commentary
Cell Therapy - Falling short of its potential?
2019   |   Pierre-Louis Joffrin
Despite major investment in the field, few cell therapies have gained regulatory approval with limited commercial success. The success of cell therapy products is defined by three factors: safety, efficacy and scalabi...