The journey of islet cell transplantation and future development

A Gamble, AR Pepper, A Bruni, AMJ Shapiro - Islets, 2018 - Taylor & Francis
Intraportal islet transplantation has proven to be efficacious in preventing severe
hypoglycemia and restoring insulin independence in selected patients with type 1 diabetes …

Generation of insulin‐secreting organoids: a step toward engineering and transplanting the bioartificial pancreas

CH Wassmer, F Lebreton, K Bellofatto… - Transplant …, 2020 - Wiley Online Library
Diabetes is a major health issue of increasing prevalence. ß‐cell replacement, by pancreas
or islet transplantation, is the only long‐term curative option for patients with insulin …

[HTML][HTML] Recombinant adeno-associated virus 8 vector in gene therapy: Opportunities and challenges

L Zhao, Z Yang, M Zheng, L Shi, M Gu, G Liu, F Miao… - Genes & …, 2024 - Elsevier
In recent years, significant breakthroughs have been made in the field of gene therapy.
Adeno-associated virus (AAV) is one of the most promising gene therapy vectors and a …

Anti-ageing gene therapy: Not so far away?

A Vaiserman, E De Falco, A Koliada, O Maslova… - Ageing research …, 2019 - Elsevier
Improving healthspan is the main objective of anti-ageing research. Currently, innovative
gene therapy-based approaches seem to be among the most promising for preventing and …

Charting the next century of insulin replacement with cell and gene therapies

HH Lau, SU Gan, H Lickert, AMJ Shapiro, KO Lee… - Med, 2021 - cell.com
The discovery of insulin a century ago changed the lives of millions of individuals suffering
from diabetes, paving the way for long-term survival. While the availability of recombinant …

Development of a liver‐specific Tet‐off AAV8 vector for improved safety of insulin gene therapy for diabetes

SU Gan, Z Fu, KC Sia, OL Kon… - The journal of gene …, 2019 - Wiley Online Library
Background Diabetes mellitus is caused by a partial or complete lack of insulin production in
the body. We have previously shown that a single injection of an adeno‐associated virus …

[HTML][HTML] Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation

KC Sia, ZY Fu, SH Mohd Rodhi, JHY Yee, K Qu… - International Journal of …, 2024 - mdpi.com
Adeno-associated viruses (AAVs) have emerged as promising tools for gene therapy due to
their safety and efficacy in delivering therapeutic genes or gene editing sequences to …

Small-molecule aptamer for regulating RNA functions in mammalian cells and animals

K Fukunaga, V Dhamodharan, N Miyahira… - Journal of the …, 2023 - ACS Publications
Synthetic riboswitches that can regulate gene expression by a small molecule recognized by
an RNA aptamer in mammalian cells have various potential applications in biotechnology …

First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model

KC Sia, SU Gan, SH Mohd Rodhi, ZY Fu, JJ Kopchick… - Gene Therapy, 2022 - nature.com
The only treatment tested for growth hormone receptor (GHR) defective Laron Syndrome
(LS) is injections of recombinant insulin-like-growth factor 1 (rhIGF1). The response is …

Modification of a constitutive to glucose-responsive liver-specific promoter resulted in increased efficacy of adeno-associated virus serotype 8-insulin gene therapy of …

KC Sia, ZY Fu, RY Calne, AC Nathwani, KO Lee… - Cells, 2020 - mdpi.com
We have previously used a hepatotropic adeno-associated viral (AAV) vector with a
modified human insulin gene to treat diabetic mice. The HLP (hybrid liver-specific promoter) …