Neurology – VJRegenMed

Nerve regeneration with polyhydroxyalkanoate-based conduits

Simon Ng — Thu, 20 Jan 2022 17:55:00 +0000

Ipsita Roy, PhD, University of Sheffield, Sheffield, UK, provides an overview of polyhydroxyalkanoate (PHA)-based nerve guidance conduits for peripheral nerve regeneration. Changes in the PHA formulation resulted in the creation of a stiff, but elastic tubular structure and in vivo studies demonstrated that the scaffold regenerated damaged nerves after a 10 week period as effectively as autologously transplanted nerves. This interview took place at the 6th World Congress of the Tissue Engineering and Regenerative Medicine International Society (TERMIS 2021).

Researching the impact of innervation on regenerating tissues

Simon Ng — Mon, 10 Jan 2022 15:38:55 +0000

Paul Wieringa, PhD, Maastricht University, Maastricht, The Netherlands, describes research on the impact innervation in nociceptors found in the peripheral nervous system in the context of nerve regeneration. Existing in vivo research on skin models highlights the importance of innervation on tissue function and repair in healing wounds and Dr Wieringa highlights the need for 3D in vitro models to apply this research in other tissues. This interview took place at the 6th World Congress of the Tissue Engineering and Regenerative Medicine International Society (TERMIS 2021).

Research barriers in neural engineering

Simon Ng — Mon, 10 Jan 2022 15:38:54 +0000

Paul Wieringa, PhD, Maastricht University, Maastricht, The Netherlands, provides an overview on issues on researching neural engineering. Culturing neural cells with other cell populations remains a challenge due to the differing need of the cell populations, where neural cells may need more glucose than other cell types. Organizing tissues in their physiological conditions to mimic in vivo state is additionally imperative to study their function and innervation. This interview took place at the 6th World Congress of the Tissue Engineering and Regenerative Medicine International Society (TERMIS 2021).

The role of stem cells in treating CNS disorders

Simon Ng — Wed, 05 Jan 2022 17:32:23 +0000

Whilst stem cells such as mesenchymal stem cells have proven to be safe in clinical trials, various issues need to be addressed to successfully develop cellular therapies for central nervous system (CNS) diseases. Anne des Rieux, PhD, Université catholique de Louvain, Louvain, Belgium, describes various barriers associated with using stem cells in CNS diseases. Large scale manufacturing of stems cells remain limited, due to logistical constraints and cell contamination. Dr des Rieux additionally highlights the need to improve delivery systems and the advantages of using allogeneic cells. This interview took place at the 6th World Congress of the Tissue Engineering and Regenerative Medicine International Society (TERMIS 2021).

Delivering drugs to the CNS with extracellular vesicles

Simon Ng — Wed, 05 Jan 2022 17:32:21 +0000

Anne des Rieux, PhD, Université catholique de Louvain, Louvain, Belgium, discusses promising research within extracellular vesicles (EVs) for the treatment of central nervous system (CNS) diseases. Whilst EVs have been previously been investigated as diagnostic tools, current research suggests EVs can play a role in drug delivery. EVs, which contain various lipids, proteins and mRNA, can cross the blood-brain barrier, which is advantageous for the delivery of drugs to the brain. This interview took place at the 6th World Congress of the Tissue Engineering and Regenerative Medicine International Society (TERMIS 2021).

Neural stem cells isolated from the CSF for a potential autologous cell therapy

admin_vjr_user — Tue, 04 Jan 2022 15:29:55 +0000

Beatriz Fernández-Muñoz, PhD, Andalusian Network of Design and Translation of Advanced Therapies, Seville, Spain, discusses the isolation of neural stem cells (NSCs) from the cerebrospinal fluid of preterm infants with intraventricular hemorrhage (IVH). Since this fluid is routinely collected during therapeutic neuroendoscopic lavage and typically discarded, there are fewer ethical concerns associated with this source of NSCs. Recent research has demonstrated that these NSCs can be expanded, cryopreserved and differentiated into neurons and glia, and do not for tumors when transplanted into mice. Ongoing pre-clinical research aims to evaluate the potential of this cell source in the development of cell therapies for preterm infants with IVH. This interview took place at the International Society for Stem Cell Research (ISSCR) 2021 Annual Meeting.

Challenges with translating gene therapies for neurodevelopmental disorders

admin_vjr_user — Fri, 31 Dec 2021 10:38:55 +0000

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, discusses the major challenges associated with the translation of gene therapies for neurodevelopmental disorders (NDDs) to the clinic. He explains the difficulty in translating therapies for NDDs from mouse models into non-human primate models and humans, given the increased brain size and complexity which support higher-level brain function. In addition, when attempting to use gene therapy approaches to restore lost gene expression seen in NDDs such as CDKL5-deficiency disorder (CDD), it can be difficult to model and predict which neurons need to be targeted in order to achieve lasting benefit in patients. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

Non-viral gene delivery & gene editing: highlights from ESGCT

admin_vjr_user — Fri, 31 Dec 2021 10:38:53 +0000

Ralf Schmid, PhD, MSCR, University of Pennsylvania, Philadelphia, PA, describes some of his highlights from the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021, which included important basic and translational research in cell and gene therapies. Dr Schmid discusses advances seen in non-viral delivery strategies for gene therapies following lessons learned from the development of the COVID-19 vaccine. He also explains that much progress has been made in gene editing during the pandemic including the development of new strategies for improving safety and efficacy of gene editing, which hold promise for the treatment of brain disorders in the future. This interview took place at the ESGCT Virtual Congress 2021.

Eladocagene exuparvovec: AAV2-mediated gene therapy for AADC deficiency

admin_vjr_user — Fri, 24 Dec 2021 10:07:54 +0000

Paul Wuh-Liang Hwu, MD, PhD, National Taiwan University Hospital, Taipei, Taiwan, describes the rationale and ongoing research into gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a very rare genetic disorder caused by mutations in the dopa decarboxylase gene leading to reduced AADC enzyme activity, resulting in delayed cognitive and speech development. Dr Hwu describes the study of eladocagene exuparvovec, a recombinant adeno-associated viral vector (AAV) serotype 2 carrying cDNA encoding the human AADC gene, which is administered via bilateral infusion to the putamen. Results demonstrate the efficacy of this treatment in improving cognition and motor development. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

LYS-GM101 AAV gene therapy for GM1 gangliosidosis

admin_vjr_user — Thu, 23 Dec 2021 16:24:52 +0000

GM1 gangliosidosis is a rare autosomal lysosomal storage disorder caused by mutations in the GLB1 gene which encodes β-galactosidase (Beta-gal), resulting in toxic accumulation of GM1 ganglioside predominantly in the central nervous system (CNS). Michael Hocquemiller, PhD, Lysogene, Paris, France, describes ongoing research into LYS-GM101, an adeno-associated virus serotype rh.10 (AAVrh.10) vector carrying GLB1 cDNA, administered via a single injection into the cisterna magna. Following promising results obtained in pre-clinical studies in mouse, cat and non-human primate disease models, an open-label two-stage adaptive design study (NCT04273269) is currently underway which will evaluate the safety and efficacy of LYS-GM101 in patients with GM1 gangliosidosis. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.