[HTML][HTML] Emerging innate biological properties of nano-drug delivery systems: A focus on PAMAM dendrimers and their clinical potential

H Kheraldine, O Rachid, AM Habib… - Advanced drug delivery …, 2021 - Elsevier
Drug delivery systems or vectors are usually needed to improve the bioavailability and
effectiveness of a drug through improving its pharmacokinetics/pharmacodynamics at an …

Applications and challenges of CRISPR-Cas gene-editing to disease treatment in clinics

W Liu, L Li, J Jiang, M Wu, P Lin - Precision clinical medicine, 2021 - academic.oup.com
Clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems
(Cas) are efficient tools for targeting specific genes for laboratory research, agricultural …

CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis

JD Gillmore, E Gane, J Taubel, J Kao… - … England Journal of …, 2021 - Mass Medical Soc
Background Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening
disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein …

[HTML][HTML] CAR-T regulatory (CAR-Treg) cells: engineering and applications

M Arjomandnejad, AL Kopec, AM Keeler - Biomedicines, 2022 - mdpi.com
Regulatory T cells are critical for maintaining immune tolerance. Recent studies have
confirmed their therapeutic suppressive potential to modulate immune responses in organ …

CRISPR/Cas9 genome editing for tissue‐specific in vivo targeting: nanomaterials and translational perspective

DK Sahel, LK Vora, A Saraswat, S Sharma… - Advanced …, 2023 - Wiley Online Library
Clustered randomly interspaced short palindromic repeats (CRISPRs) and its associated
endonuclease protein, ie, Cas9, have been discovered as an immune system in bacteria …

[HTML][HTML] Advance trends in targeting homology-directed repair for accurate gene editing: An inclusive review of small molecules and modified CRISPR-Cas9 systems

F Shams, H Bayat, O Mohammadian, S Mahboudi… - BioImpacts …, 2022 - ncbi.nlm.nih.gov
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Introduction: Clustered regularly interspaced short palindromic repeat and its associated …

[HTML][HTML] CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa

WH Wu, YT Tsai, IW Huang, CH Cheng, CW Hsu… - Molecular Therapy, 2022 - cell.com
Mutations in rhodopsin (RHO) are the most common causes of autosomal dominant retinitis
pigmentosa (adRP), accounting for 20% to 30% of all cases worldwide. However, the high …

[HTML][HTML] A potential paradigm in CRISPR/Cas systems delivery: at the crossroad of microalgal gene editing and algal-mediated nanoparticles

S Feng, X Xie, J Liu, A Li, Q Wang, D Guo, S Li… - Journal of …, 2023 - Springer
Microalgae as the photosynthetic organisms offer enormous promise in a variety of
industries, such as the generation of high-value byproducts, biofuels, pharmaceuticals …

Gene therapy for neurotransmitter‐related disorders

WS Chu, J Ng, SN Waddington… - Journal of Inherited …, 2024 - Wiley Online Library
Inborn errors of neurotransmitter (NT) metabolism are a group of rare, heterogenous
diseases with predominant neurological features, such as movement disorders, autonomic …

[HTML][HTML] Unanswered questions in the regulation and function of the duplicated α7 nicotinic receptor gene CHRFAM7A

S Leonard, R Benfante - Pharmacological Research, 2023 - Elsevier
The α7 nicotinic receptor (α7 nAChR) is an important entry point for Ca 2+ into the cell,
which has broad and important effects on gene expression and function. The gene …