Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs

SA Dilliard, DJ Siegwart - Nature Reviews Materials, 2023 - nature.com
Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …

ApoE in Alzheimer's disease: pathophysiology and therapeutic strategies

AC Raulin, SV Doss, ZA Trottier, TC Ikezu, G Bu… - Molecular …, 2022 - Springer
Alzheimer's disease (AD) is the most common cause of dementia worldwide, and its
prevalence is rapidly increasing due to extended lifespans. Among the increasing number of …

The current landscape of nucleic acid therapeutics

JA Kulkarni, D Witzigmann, SB Thomson… - Nature …, 2021 - nature.com
The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Tumor immune contexture is a determinant of anti-CD19 CAR T cell efficacy in large B cell lymphoma

N Scholler, R Perbost, FL Locke, MD Jain, S Turcan… - Nature medicine, 2022 - nature.com
Axicabtagene ciloleucel (axi-cel) is an anti-CD19 chimeric antigen receptor (CAR) T cell
therapy approved for relapsed/refractory large B cell lymphoma (LBCL) and has treatment …

Carbon dots: a new type of carbon-based nanomaterial with wide applications

J Liu, R Li, B Yang - ACS Central Science, 2020 - ACS Publications
Carbon dots (CDs), as a new type of carbon-based nanomaterial, have attracted broad
research interest for years, because of their diverse physicochemical properties and …

[HTML][HTML] Betibeglogene Autotemcel Gene Therapy for Non–β00 Genotype β-Thalassemia

F Locatelli, AA Thompson, JL Kwiatkowski… - … England Journal of …, 2022 - Mass Medical Soc
Background Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-
thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells …

Nanodelivery of nucleic acids

BB Mendes, J Conniot, A Avital, D Yao… - Nature reviews …, 2022 - nature.com
There is growing need for a safe, efficient, specific and non-pathogenic means for delivery of
gene therapy materials. Nanomaterials for nucleic acid delivery offer an unprecedented …

Engineering adeno-associated virus vectors for gene therapy

C Li, RJ Samulski - Nature Reviews Genetics, 2020 - nature.com
Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

[图书][B] Wicked problems in public policy: Understanding and responding to complex challenges

BW Head - 2022 - library.oapen.org
This is an open access book. This book offers the first overview of the 'wicked problems'
literature, often seen as complex, open-ended, and intractable, with both the nature of the …

Deep diversification of an AAV capsid protein by machine learning

DH Bryant, A Bashir, S Sinai, NK Jain, PJ Ogden… - Nature …, 2021 - nature.com
Modern experimental technologies can assay large numbers of biological sequences, but
engineered protein libraries rarely exceed the sequence diversity of natural protein families …