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Here are some of the biggest medical advances in 2023.
New treatments include the first CRISPR gene-editing therapy, an Alzheimer’s drug and RSV vaccines
In March, the U.S. Food and Drug Administration said the nasal spray Narcan, which can reverse the effects of an opioid overdose, could be sold over the counter.
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By Erin Wayman
December 14, 2023 at 7:00 am
Weight-loss drugs stole much of the spotlight in 2023, but these medical advances treating other conditions are also worthy of attention ( SN: 12/13/23 ).
Green light for CRISPR gene editing
On December 8, the U.S. Food and Drug Administration approved the world’s first CRISPR/Cas9 gene-editing therapy ( SN: 12/8/23 ). The treatment, called Casgevy, targets sickle cell disease by helping patients produce healthy hemoglobin. In people with the disease, hemoglobin is abnormal, causing red blood cells to become hard and crescent shaped, which can block blood flow. By March 2024, the FDA will decide whether the same therapy can be used to treat beta-thalassemia, a disorder that reduces hemoglobin production.
Slowing down Alzheimer’s
The Alzheimer’s drug lecanemab (brand name Leqembi) won full FDA approval in July. Like the drug aducanumab approved in 2021, lecanemab removes the amyloid plaques that build up in the brains of people with Alzheimer’s. The drug doesn’t stop the disease, but in a clinical trial, lecanemab slowed cognitive decline by about 30 percent over 18 months compared with a placebo ( SN: 8/12/23, p. 9 ).
A gene therapy for muscular dystrophy
In June, the FDA approved the first gene therapy for children with Duchenne muscular dystrophy. Due to a faulty gene, people with this muscle-wasting disease don’t make the protein dystrophin, which helps keep muscle cells intact. The therapy helps the body produce a version of the missing protein ( SN: 6/22/23 ).
Guarding against RSV
Several ways to protect against respiratory syncytial virus arrived this year. In May, the FDA approved the first RSV vaccine, called Arexvy, in the United States , for adults age 60 and older ( SN: 6/17/23, p. 8 ), and then in August, a vaccine for pregnant people , called Abrysvo ( SN: 8/25/23 ). A monoclonal antibody — a lab-made antibody that mimics immune system proteins — won approval in July to protect children 2 and younger from the virus, which sends as many as 80,000 young children to U.S. hospitals each year (S N: 4/27/23 ). But in October, limited supplies of the therapy led the U.S. Centers for Disease Control and Prevention to recommend reserving it for babies at highest risk for complications from RSV.
A pill for postpartum depression
Until August, the only medication in the United States specifically targeting postpartum depression required a 60-hour intravenous infusion in a hospital ( SN: 3/22/19 ). With FDA approval of zuranolone (brand name Zurzuvae), those afflicted with postpartum depression can take an oral medication at home and experience improvement in as little as three days .
Birth control, no prescription required
In July, the FDA ruled that the oral contraceptive norgestrel, first approved in 1973, be available without a prescription. It’s the first OTC daily birth control pill in the United States. Some public health experts argue that reducing barriers to contraception is especially important to reproductive autonomy now that state bans have limited access to abortion ( SN: 5/19/23 ).
A shot against chikungunya
The chikungunya virus can cause fever and severe joint pain, and be fatal to newborns. In November, the FDA approved the first vaccine against the virus , which is transmitted by mosquitoes. The virus is most prevalent in tropical regions, but the FDA warns that it’s spreading to new parts of the globe.
Narcan over the counter
The nasal spray Narcan, aka naloxone, can reverse the effects of an opioid overdose within minutes. In March, the FDA ruled this life-saving drug can be sold over the counter . Officials hope that easier access to Narcan can help fight the opioid epidemic, which claimed the lives of nearly 645,000 people from 1999 to 2021 due to overdoses.
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Collection 06 March 2024
The Top 25 Health Sciences Articles of 2023
We are pleased to share with you the 25 most downloaded Nature Communications articles* in health sciences published in 2023. (Please note we have a separate collection for the Top 25 COVID-19 papers .) Featuring authors from around the world, these papers highlight valuable research from an international community.
Browse all Top 25 subject area collections .
*Data obtained from SN Insights (based on Digital Science's Dimensions) and have been normalised to account for articles published later in the year.
Top Articles
Dietary circadian rhythms and cardiovascular disease risk in the prospective nutrinet-santé cohort.
The relation between daily meal and fasting timing with cardiovascular disease incidence remains unclear. Here, authors show that a later daily meal timing is associated with increased cardiovascular disease risk, especially in women. This study suggests that adopting earlier daily eating patterns may be beneficial for cardiovascular prevention.
- Anna Palomar-Cros
- Valentina A. Andreeva
- Bernard Srour
On-demand male contraception via acute inhibition of soluble adenylyl cyclase
Half of all pregnancies are unintended; thus, existing family planning options are inadequate. This proof-of-concept study validates an on-demand contraception strategy for men, showing high effectiveness in quickly and temporarily reducing male fertility in mice.
- Melanie Balbach
- Thomas Rossetti
- Lonny R. Levin
Identification of 5-HT 2A receptor signaling pathways associated with psychedelic potential
Serotonin 5-HT 2A receptor signaling mechanisms associated with predicting psychedelic potential remain elusive. Using 5-HT 2A -selective β-arrestin-biased ligands, here the authors show that a threshold level of 5-HT 2A -Gq efficacy and not β-arrestin recruitment is associated with psychedelic potential.
- Jason Wallach
- Andrew B. Cao
- John D. McCorvy
Mid-old cells are a potential target for anti-aging interventions in the elderly
In this study, the authors introduce the concept of a unique cellular subtype within the organic stroma, which does not conform to a typical young or senescent but is significantly associated with age-related organic dysfunction among the elderly.
- Young Hwa Kim
- Young-Kyoung Lee
- Tae Jun Park
Injectable and biodegradable piezoelectric hydrogel for osteoarthritis treatment
The use of biomaterial scaffolds-based cartilage grafts could potentially innovate the Osteoarthritis (OA) treatment, but has been limited by toxicity concerns and invasive surgical procedures. Here, the authors report an injectable and biodegradable piezoelectric hydrogel with ultrasound activation to offer a minimally invasive approach for OA treatment.
- Tra Vinikoor
- Godwin K. Dzidotor
- Thanh D. Nguyen
Host-diet-gut microbiome interactions influence human energy balance: a randomized clinical trial
The gut microbiome is causally linked to body weight in preclinical models. Here, in a controlled feeding study, the authors show that greater delivery of gut-microbiome fermentable dietary substrates to the colon leads to a net negative energy balance that is accompanied by robust microbial and host responses.
- Karen D. Corbin
- Elvis A. Carnero
- Steven R. Smith
Respiration modulates sleep oscillations and memory reactivation in humans
The memory function of sleep relies on the coordination of slow oscillations and spindles. Here the authors show that respiration is associated with the emergence and interplay of these sleep rhythms, and that this coupling is linked to memory reactivation.
- Thomas Schreiner
- Marit Petzka
- Bernhard P. Staresina
Delayed gut microbiota maturation in the first year of life is a hallmark of pediatric allergic disease
Here, using participants in the CHILD birth cohort, the authors reveal that impaired 1-year microbiota maturation may be universal to 5-year pediatric allergies, mediated by functional and metabolic imbalances of compromised mucous integrity, elevated oxidative activity, decreased fermentation, and elevated trace amines.
- Courtney Hoskinson
- Darlene L. Y. Dai
- Stuart E. Turvey
Platelet-derived exerkine CXCL4/platelet factor 4 rejuvenates hippocampal neurogenesis and restores cognitive function in aged mice
Exercise has positive effects on the brain during aging. Here the authors show that in mice, platelet-released exerkine PF4 mediates the effects of exercise on the brain.
- Odette Leiter
- David Brici
- Tara L. Walker
mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy
Large genes require dual adeno-associated viral (AAV) vectors for in vivo delivery/expression, but current methods have limitations. Here the authors develop and functionally evaluate REVeRT, an efficient and flexible dual AAV vector technology based on reconstitution via mRNA trans-splicing.
- Lisa Maria Riedmayr
- Klara Sonnie Hinrichsmeyer
- Elvir Becirovic
Biofilm microenvironment triggered self-enhancing photodynamic immunomodulatory microneedle for diabetic wound therapy
The treatment of diabetic wounds tends to be hindered by complex wound environments, and the critical role of the microenvironment in the chronic diabetic wounds has not been explored for therapeutic development. Here, the authors develop a wound microenvironment-responsive microneedle bandage to achieve self-enhanced, catabolic and dynamic therapy of chronic wounds.
- Xiaowei Zeng
Ultra-durable cell-free bioactive hydrogel with fast shape memory and on-demand drug release for cartilage regeneration
Achieving successful in vivo cartilage regeneration remains challenging. Here they present a cell-free, multiple hydrogen-bond crosslinked hydrogel loaded with tannic acid and Kartogenin with ultra-durable mechanical properties and stage-dependent drug release behavior to promote cartilage regeneration.
- Yuxuan Yang
- Xiaodan Zhao
Cancer-associated fibroblasts actively compress cancer cells and modulate mechanotransduction
Cancer-associated fibroblasts (CAFs) can produce ECM and form a physical barrier around the tumour. Here, the authors show in transgenic mouse models and in vitro systems that CAFs are able to actively compress cancer cells using actomyosin contractility and this leads to a modulation of cancer cell mechanosensing and tumour reorganisation.
- Jorge Barbazan
- Carlos Pérez-González
- Danijela Matic Vignjevic
Essential transcription factors for induced neuron differentiation
Using integrative multi-omics and CRISPR knock-out of all ~1,900 transcription factors, the authors identify essential transcription factors required for Neurogenin-driven differentiation of human cortical neurons.
- Görkem Garipler
- Neville E. Sanjana
Differential responses of the gut microbiome and resistome to antibiotic exposures in infants and adults
Knowledge on how the gut microbiome and resistome responds to antibiotics across age remains limited. Here, using metagenomics data from Danish infants and young adults, the authors show that antibiotics have a more lasting impact on adults compared to infants.
- Asker Brejnrod
- Søren Johannes Sørensen
Cell type-specific delivery by modular envelope design
Targetable delivery vectors for genetic cargo are needed. Here the authors report a modular platform with separate fusion and targeting components—Delivery to Intended REcipient Cells Through Envelope Design (DIRECTED)—and show cell type-specific delivery.
- Daniel Strebinger
- Chris J. Frangieh
Characterizations of a neutralizing antibody broadly reactive to multiple gluten peptide:HLA-DQ2.5 complexes in the context of celiac disease
Targeting gluten antigens presents a plausible therapy option for celiac disease. Here the authors generate and characterize a broadly neutralizing antibody recognizing more than 25 gluten peptide:HLA-DQ2.5 complexes, with structural analyses implicating its mode of interaction, and with mouse in vivo studies supporting its therapeutic feasibility.
- Yuri Ikawa-Teranishi
- Tomoyuki Igawa
Identification of indocyanine green as a STT3B inhibitor against mushroom α-amanitin cytotoxicity
There is currently no specific antidote for death cap mushroom poisoning treatment. Here, the authors identify STT3B as a druggable target and show that indocyanine green is a STT3B inhibitor that can block α-amanitin toxicity in cell lines, liver organoids and mice.
- Arabella H. Wan
- Qiao-Ping Wang
Associations of timing of physical activity with all-cause and cause-specific mortality in a prospective cohort study
There is a growing interest in the role of timing of physical activity (PA) in improving health. Here, using a large-scale cohort study, the authors show that moderate-to-vigorous PA at the optimal time of day robustly predicts lower mortality risk and may maximize the beneficial effect of PA.
- Hongliang Feng
- Jihui Zhang
Mitochondrial malfunction and atrophy of astrocytes in the aged human cerebral cortex
How aging affects the brain active milieu remains unknown. Here, the authors reveal atrophy and mitochondrial malfunction of astrocytes but not neurons in older human neocortex.
- Alexander Popov
- Nadezda Brazhe
- Alexey Semyanov
A class of secreted mammalian peptides with potential to expand cell-cell communication
The cells of our bodies use chemical signals to talk with each other. Here the authors describe a class of signaling molecules called “capped peptides” that may mediate cell-cell communication. Unlike other peptides, capped peptides have unique chemical modifications which make them potentially more active and stable.
- Amanda L. Wiggenhorn
- Hind Z. Abuzaid
- Jonathan Z. Long
Human and mouse neutrophils share core transcriptional programs in both homeostatic and inflamed contexts
Difficulties can be encountered when translating research between cells from animals and humans because of gene expression differences. Here the authors perform an integrative transcriptomic analysis from human and mouse neutrophils and identify a core inflammation program shared across inflamed contexts.
- Nicolaj S. Hackert
- Felix A. Radtke
- Ricardo Grieshaber-Bouyer
Open access repository-scale propagated nearest neighbor suspect spectral library for untargeted metabolomics
Interpreting untargeted mass spectrometry (MS) data is challenging due to incomplete reference libraries. Here, the authors created the nearest neighbor suspect spectral library from largescale public MS data, significantly enhancing the ability to hypothesize structures for unknown mass spectra.
- Wout Bittremieux
- Nicole E. Avalon
- Pieter C. Dorrestein
Temporal morphogen gradient-driven neural induction shapes single expanded neuroepithelium brain organoids with enhanced cortical identity
PSC-brain organoids are typically formed by static medium switches. Here, authors show that a temporal morphogen gradient during neural induction allows the formation of well-specified cortical organoids with a self-organized single neuroepithelium.
- Anna Pagliaro
- Roxy Finger
- Benedetta Artegiani
High-resolution neural recordings improve the accuracy of speech decoding
Previous work has shown speech decoding in the human brain for the development of neural speech prostheses. Here the authors show that high density µECoG electrodes can record at micro-scale spatial resolution to improve neural speech decoding.
- Suseendrakumar Duraivel
- Shervin Rahimpour
- Gregory B. Cogan
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A new implant tested in animals reverses drug overdoses. In pigs, the device detected overdoses and administered naloxone. It could also alert emergency services to respond. By Anna Gibbs 1 hour ...
Here are some of the biggest medical advances in 2023. New treatments include the first CRISPR gene-editing therapy, an Alzheimer’s drug and RSV vaccines. In March, the U.S. Food and Drug ...
2021 Top 25 Health Sciences Articles. We are pleased to share with you the 25 most downloaded Nature Communications articles* in health sciences published in 2021. (Please note we have...
PubMed Central ® (PMC) is a free full-text archive of biomedical and life sciences journal literature at the U.S. National Institutes of Health's National Library of Medicine (NIH/NLM) About PMC. Discover a digital archive of scholarly articles, spanning centuries of scientific research. User Guide.
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Browse the 25 most downloaded Nature Communications articles in health sciences published in 2023.