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A groundbreaking advancement in gene editing heralds a new era for immunotherapy, addressing the longstanding challenges of integrating large DNA sequences into primary human T cel...
Beverly Davidson, PhD, chief scientific strategy officer at Children's Hospital of Philadelphia, shares her research including a novel gene therapy approach for Huntington's diseas...
Advanced non-viral gene delivery systems are expanding the range of indications and therapy modalities possible for the new generation of genetic medicines. The post Going Non-Vira...
Gene editing represents one of the most transformative advances in modern medicine, offering the potential to correct genetic diseases at their source. However, a major obstacle in...
As more products reach the market, therapy developers partner with researchers and regulators to deliver N-of-1 treatments to patients. The post Top 10 Best Selling Gene Therapies...
A new platform based on genome-wide screening systemically identifies which genes drive or block particle assembly to engineer cells that yield more potent delivery vehicles. The...
In a groundbreaking advancement for gene therapy, researchers have unveiled two novel bioengineered AAV8 vectors—HMR-001 and its codon-optimized counterpart HMR-001z—that demonstra...
An experimental gene therapy called SynCav1 can protect the central nervous system from the cognitive decline linked to TDP-43-related proteinopathy, a primary driver of FTD, ALS,...
Researchers here describe a novel approach to encourage greater regeneration in heart tissue following the injury and lost function incurred during a heart attack. Their work falls...
In a groundbreaking advancement in the field of gene therapy, researchers have demonstrated a significant clinical response following systemic administration of adeno-associated vi...
Tinkering with the genetic code of a living human is risky. The post Gene Therapy Causes Patient to Grow Alarming Tumor appeared first on Futurism.
What’s most exciting about CRISPR is its potential to shift medicine from managing disease to directly correcting its root cause. End-to-end pipelines and regulatory advances aim t...
The therapy is part of a plan to develop gene therapies that can be efficiently delivered at lower doses, enabling them to be used against more than just “ultra-rare” conditions.
MONDAY, April 27, 2026 — In a historic leap for medical science, the U.S. Food and Drug Administration (FDA) has greenlit the first-ever gene therapy to treat a rare form of inheri...
A multicenter study found a one-time treatment targeting the OTOF gene restored hearing in most participants, with effects lasting up to 2.5 years.
Travis Smith was a very serious baby. Born completely unable to hear, his mother, Sierra, struggled to see his personality shine through his mute world.
The modular plug-and-play protein adapter developed in collaboration with Menachem Rubenstein, PhD, of the Weizmann Institute allows drug developers to cap and retarget existing le...
A new study improves gene editing delivery by engineering the human cells that produce virus-like particles, boosting the potency of these key therapeutic vehicles.
A Phase I/II clinical trial shows that a stem cell transplant that removes CD33 from donor cells using CRISPR can prevent cancer recurrence. The post Acute Myeloid Leukemia Therapy...
A new gene‑editing strategy programs hematopoietic stem cells to produce therapeutic antibodies and other proteins. The approach generates long‑lasting immunity in mice and could p...
Leronlimab is an antiviral HIV drug that targets and blocks the CCR5 receptor, thus blocking HIV’s ability to invade immune cells. Most macaques that produced sufficient number of...
In a groundbreaking study that could redefine treatment paradigms for spinal muscular atrophy (SMA), researchers have released a comprehensive long-term comparative analysis of ade...
In a landmark advance for genetic medicine, researchers have unveiled a groundbreaking approach to correcting a cryptic splice mutation responsible for cardiac-type Fabry disease....
New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options.
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