Correction to: A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

Urits, Ivan; Swanson, Douglas R.; Swett, Michael C; Patel, Arpan; Berardino, Kevin; Amgalan, Ariunzaya; Berger, Amnon A; Kassem, Hisham; Kaye, Alan D.; Viswanath, Omar

doi:10.1007/s40120-020-00228-x

Cited by 7 publications

(3 citation statements)

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“…RNAi-mediated mechanisms of cell fate regulation combined with nanomedicine are gaining traction as avenues to develop innovative molecular therapeutics. Thus far, three siRNA-based therapeutics have been approved by the Food and Drug Administration, Patisiran, Givosiran, and Lumasiran 57 , 58 with multiple others currently in clinical trials. 59 These promising developments open the door for new therapeutic approaches based on reconstituting tumor-suppressive miRNAs or inhibiting oncogenic miRNAs as means to normalize dysregulated molecular networks, inhibit tumor growth, and enhance the effects of current standards-of-care.…”

Section: Discussionmentioning

confidence: 99%

Sox2 induces glioblastoma cell stemness and tumor propagation by repressing TET2 and deregulating 5hmC and 5mC DNA modifications

López-Bertoni

Johnson

Rui

et al. 2022

Sig Transduct Target Ther

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DNA methylation is a reversible process catalyzed by the ten–eleven translocation (TET) family of enzymes (TET1, TET2, TET3) that convert 5-methylcytosine (5mC) to 5-hydroxymethylcytosine (5hmC). Altered patterns of 5hmC and 5mC are widely reported in human cancers and loss of 5hmC correlates with poor prognosis. Understanding the mechanisms leading to 5hmC loss and its role in oncogenesis will advance the development of epigenetic-based therapeutics. We show that TET2 loss associates with glioblastoma (GBM) stem cells and correlates with poor survival of GBM patients. We further identify a SOX2:miR-10b-5p:TET2 axis that represses TET2 expression, represses 5hmC, increases 5mC levels, and induces GBM cell stemness and tumor-propagating potential. In vivo delivery of a miR-10b-5p inhibitor that normalizes TET2 expression and 5hmC levels inhibits tumor growth and prolongs survival of animals bearing pre-established orthotopic GBM xenografts. These findings highlight the importance of TET2 and 5hmC loss in Sox2-driven oncogenesis and their potential for therapeutic targeting.

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Section: Discussionmentioning

confidence: 99%

Sox2 induces glioblastoma cell stemness and tumor propagation by repressing TET2 and deregulating 5hmC and 5mC DNA modifications

López-Bertoni

Johnson

Rui

et al. 2022

Sig Transduct Target Ther

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“…LNPs, as one of the most studied nanocarriers, undergo rapid technological evolution and get significant advances. For instance, the first siRNA drug, Onpattro™ (a new small interfering RNA (siRNA) LNP), was launched in 2018 for treating hATTR polyneuropathy, signifying a milestone advancement in LNP technology [ 82 ]. The key to Onpattro™ success is the development of ionizable cationic lipids.…”

Section: Liposomes and Lipid Nanoparticles (Lnps)mentioning

confidence: 99%

Approved Nanomedicine against Diseases

Jia

Jiang

et al. 2023

Pharmaceutics

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Nanomedicine is a branch of medicine using nanotechnology to prevent and treat diseases. Nanotechnology represents one of the most effective approaches in elevating a drug‘s treatment efficacy and reducing toxicity by improving drug solubility, altering biodistribution, and controlling the release. The development of nanotechnology and materials has brought a profound revolution to medicine, significantly affecting the treatment of various major diseases such as cancer, injection, and cardiovascular diseases. Nanomedicine has experienced explosive growth in the past few years. Although the clinical transition of nanomedicine is not very satisfactory, traditional drugs still occupy a dominant position in formulation development, but increasingly active drugs have adopted nanoscale forms to limit side effects and improve efficacy. The review summarized the approved nanomedicine, its indications, and the properties of commonly used nanocarriers and nanotechnology.

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“…The interactions mediate the cellular internalization of nanoparticles along with siRNA and their release in the cytoplasm ( Jayaraman et al, 2012 ; Lee et al, 2022 ). DLin-MC3-DMA [(6Z,9Z,28Z,31Z)-heptatriacont-6,9,28,31-tetraene-19-yl 4-(dimethylamino) butanoate], ALC-0315 {[(4-hydroxybutyl) azanediyl]di(hexane-6,1-diyl) bis(2-hexyldecanoate)}, and SM-102 {1-Octylnonyl 8-[(2-hydroxyethyl) (6-oxo-6 (undecyloxy)hexyl)amino]-octanoate} are clinically approved lipids, among which ALC-0315 and SM-102 have been applied for mRNA delivery, and DLin-MC3-DMA (MC3) has been used for siRNA delivery for treating transthyretin amyloidosis ( Suzuki and Ishihara, 2021 ; Urits et al, 2021 ; Saadati et al, 2022 ). The helper lipids are generally phospholipids such as DOPE (dioleoylphosphatidylethanolamine) and DSPC (distearoylphosphatidylcholine) and are used for the stability of LNPs and for aiding the endosomal release ( Hou et al, 2021 ).…”

Section: Nanoparticles (Nps) Used For the Sirna Therapy Of Covid-19mentioning

confidence: 99%

Potential of siRNA in COVID-19 therapy: Emphasis on in silico design and nanoparticles based delivery

Fopase

Panda

Rajendran

et al. 2023

Front. Bioeng. Biotechnol.

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Small interfering RNA (siRNA)-mediated mRNA degradation approach have imparted its eminence against several difficult-to-treat genetic disorders and other allied diseases. Viral outbreaks and resulting pandemics have repeatedly threatened public health and questioned human preparedness at the forefront of drug design and biomedical readiness. During the recent pandemic caused by the SARS-CoV-2, mRNA-based vaccination strategies have paved the way for a new era of RNA therapeutics. RNA Interference (RNAi) based approach using small interfering RNA may complement clinical management of the COVID-19. RNA Interference approach will primarily work by restricting the synthesis of the proteins required for viral replication, thereby hampering viral cellular entry and trafficking by targeting host as well as protein factors. Despite promising benefits, the stability of small interfering RNA in the physiological environment is of grave concern as well as site-directed targeted delivery and evasion of the immune system require immediate attention. In this regard, nanotechnology offers viable solutions for these challenges. The review highlights the potential of small interfering RNAs targeted toward specific regions of the viral genome and the features of nanoformulations necessary for the entrapment and delivery of small interfering RNAs. In silico design of small interfering RNA for different variants of SARS-CoV-2 has been discussed. Various nanoparticles as promising carriers of small interfering RNAs along with their salient properties, including surface functionalization, are summarized. This review will help tackle the real-world challenges encountered by the in vivo delivery of small interfering RNAs, ensuring a safe, stable, and readily available drug candidate for efficient management of SARS-CoV-2 in the future.

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Correction to: A Review of Patisiran (ONPATTRO®) for the Treatment of Polyneuropathy in People with Hereditary Transthyretin Amyloidosis

Abstract: The middle initial of one of the authors, Alan Kaye, was missed in the original publication.The complete name should read as Alan D. Kaye.The original article has been corrected.

Cited by 7 publications

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Sox2 induces glioblastoma cell stemness and tumor propagation by repressing TET2 and deregulating 5hmC and 5mC DNA modifications

Sox2 induces glioblastoma cell stemness and tumor propagation by repressing TET2 and deregulating 5hmC and 5mC DNA modifications

Approved Nanomedicine against Diseases

Potential of siRNA in COVID-19 therapy: Emphasis on in silico design and nanoparticles based delivery

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