Animal Research Stories

Browse news and features about how animal models are enabling discoveries at Harvard Medical School and beyond, and meet some of the people who work with animals in our community.

Connie Cepko sits in a chair in her office petting her dog, Stewie
Video: Stephanie Dutchen and Rick Groleau

The Journey toward a Gene Therapy for Retinitis Pigmentosa

Animals can play crucial roles in helping researchers understand disease and develop new treatments. Without mice, Harvard Medical School geneticist Connie Cepko wouldn’t be on the cusp of discovering whether her team has developed the first safe and effective gene therapy for people with a form of genetic blindness called retinitis pigmentosa.

An estimated 1.5 million people worldwide live with retinitis pigmentosa, which starts with loss of night vision and usually progresses to loss of color, peripheral, and/or central vision. There is currently no cure and no way to stop the disease from advancing.

In this video, Cepko shares her team's approach to developing a gene therapy that aims to preserve vision in people with RP and certain other forms of genetic blindness, including some cases of macular degeneration, by counteracting damage to the retina caused by any of hundreds of different genetic mutations.

More news and features

Gary Ruvkun with daughter Victoria Ruvkun (left), dog Barnaby, and wife Natasha Staller the morning of the announcement at their home in Newton.
Harvard Medical School Scientist Gary Ruvkun Receives Nobel Prize for Discovery of microRNA
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Ruvkun shares award with Victor Ambros for elucidating a fundamental principle of gene activity regulation
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Mouse study illuminates immune-evading mechanism, suggests how to turn the tables
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Creating the Next Generation of mRNA Vaccines
July 29, 2024

New tech promises longer and more potent protection with smaller doses and fewer side effects
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Meet the Brain Cells that May Help Us Adapt to Changes in Day Length
July 17, 2024

Study illuminates circadian rhythms, psychiatric disorders with seasonal flare-ups
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Exploring Our Sense of Touch from Every Angle
June 11, 2024

Harvard Medical School researchers are illuminating one of the most mysterious — and most essential — senses
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A Clue Into How We See a Stable World Despite Moving Eyes
April 29, 2024

Macaque study illuminates how brain perceives static images amid constant eye movements
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Repurposed Cancer Drugs May Improve Tuberculosis Treatment
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Drug combination enhanced delivery of antibacterial medications in rabbits
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Reflections on Neurobiology, Past and Present
March 26, 2024

Department co-founder shares thoughts on how far the field has come — and what’s next
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In a First, Genetically Edited Pig Kidney Is Transplanted Into Human
March 21, 2024

Procedure marks milestone in quest to provide more organs to patients in need
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How Does the Brain Make Decisions?
February 21, 2024

Mouse study provides insights into communication between neurons during decision-making
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How the Brain’s Compass Guides the Body
February 7, 2024

A study in fruit flies reveals how the brain’s compass and steering regions make course corrections
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HIV Drugs Show Promise in Averting Complications from Bacterial Infections
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Study in mice finds a trigger for devastating condition, hints at possible treatment
white woman wearing a backpack, viewed from behind, spraying insect repellant onto her bare forearm in a field next to some woods
Worm Study Raises Concern About DEET’s Effect on Reproduction
January 4, 2024

Findings suggest need to consider reproductive health alongside prevention of insect-borne diseases
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What Happens in the Brain While Daydreaming?
December 13, 2023

Observations in mice hint at role of daydreams in remodeling the brain
a runner's legs exercising
Some Benefits of Exercise Stem from the Immune System
November 3, 2023

Immune cells mobilized in the muscles during exercise fend off inflammation and boost endurance
A shot of Jessica Chaikof from the chest up, wearing a pink shirt and glasses
Pursuing Three Gene Therapies for Rare Inherited Disease
October 19, 2023

Researchers hope at least one will treat progressive blindness caused by Usher syndrome type 1F
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How the Heart Starts Beating
September 27, 2023

Researchers discover that heart cells in developing zebrafish start beating suddenly and all at once
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How Neurons Grow Comfortable in Their Own Skin
August 23, 2023

Sensory neurons develop customized nerve endings based on cues from the skin, study in mice suggests
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Scientists Identify Cells Involved in Correcting Errors During Spatial Navigation
July 19, 2023

Neurons become active when mice exploring a maze correct after making a wrong turn
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Researchers Uncover Signal Needed for Blood-Brain Barrier
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The signal, produced by neurons, helps the barrier form and maintain its protective properties
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When It Comes to Immunity, You Are What You Eat
June 28, 2023

Mouse study shows how diet altered by gut microbes spurs development of immune cells
Untangling the Mystery of Sleep
April 11, 2023

Recent sleep research reveals unexpected connections between the brain and gut
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How the Brain Senses Infection
March 8, 2023

Researchers discovered specific airway neurons in mice that alert the brain about the flu
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The Case for Female Mice in Neuroscience Research
March 7, 2023

Findings reveal female mice have more stable exploratory behavior than male mice