Evidence map›Paper›PMID 32024082›Full record

ReviewInternational journal of molecular sciences2020

Targeting Macromolecules to CNS and Other Hard-to-Treat Organs Using Lectin-Mediated Delivery.

Walter Acosta, Carole L Cramer

Abstract readReview
In one paragraph

Review in International journal of molecular sciences, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Article
  4. Article
  5. Article
  6. Article
  7. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors.

Walter AcostaBioStrategies LC, PO Box 2428, State University, Jonesboro, AR 72467, USA.ORCID 0000-0002-5525-306X
Carole L CramerBioStrategies LC, PO Box 2428, State University, Jonesboro, AR 72467, USA.ORCID 0000-0002-4133-2066

Funding

Enzyme replacement therapy for GM1 gangliosidosis lysosomal rare diseaseR44NS084565 · NINDS · BIOSTRATEGIES, LC · PI ACOSTA, WALTER, CRAMER, CAROLE L. · 2017 to 2018
$2.4M
Improving MPS I ERT Efficacy through Lectin-Mediated DeliveryR44NS102086 · NINDS · BIOSTRATEGIES, LC · PI ACOSTA, WALTER, CRAMER, CAROLE L. · 2017 to 2018
$2.4M
Enzyme replacement therapy for Sanfilippo A lysosomal rare diseaseR44NS083230 · NINDS · BIOSTRATEGIES, LC · PI CRAMER, CAROLE L., D'AZZO, ALESSANDRA · 2015 to 2016
$1.5M
NINDS NIH HHS 1R44NS102086-01NINDS NIH HHS 2R44NS083230-02A1NINDS NIH HHS 2R44NS084565-02
6 · The paper itself

Abstract

The greatest challenges for therapeutic efficacy of many macromolecular drugs that act on intracellular are delivery to key organs and tissues and delivery into cells and subcellular compartments. Transport of drugs into critical cells associated with disease, including those in organs protected by restrictive biological barriers such as central nervous system (CNS), bone, and eye remains a significant hurdle to drug efficacy and impacts commercial risk and incentives for drug development for many diseases. These limitations expose a significant need for the development of novel strategies for macromolecule delivery. RTB lectin is the non-toxic carbohydrate-binding subunit B of ricin toxin with high affinity for galactose/galactosamine-containing glycolipids and glycoproteins common on human cell surfaces. RTB mediates endocytic uptake into mammalian cells by multiple routes exploiting both adsorptive-mediated and receptor-mediated mechanisms. In vivo biodistribution studies in lysosomal storage disease models provide evidence for the theory that the RTB-lectin transports corrective doses of enzymes across the blood-brain barrier to treat CNS pathologies. These results encompass significant implications for protein-based therapeutic approaches to address lysosomal and other diseases having strong CNS involvement.

Indexed as

Drug Delivery SystemsAnimalsBiological TransportBlood-Brain BarrierCentral Nervous SystemHumansLectinsMacromolecular SubstancesTissue DistributionLectinsMacromolecular SubstancesBBBenzyme replacement therapy (ERT)lectinlysosomal storage diseases, CNSmacromolecule transportRTB

Identifiers

PMID32024082
PMCPMC7037663

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.